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The document provides information about a Phase 2 clinical trial, NCT06623110, investigating the use of RP2 Injection in patients with high-risk oral potentially malignant disorders (OPD). The trial aims to evaluate the safety, tolerability, and potential efficacy of RP2 Injection in treating OPD and preventing oral cancer. The study is not yet recruiting participants and is expected to last for up to 2 years, with an estimated completion date of January 1, 2028. The eligibility criteria for participants include specific diagnostic criteria for high-risk OPD and certain health requirements. The study is being conducted by the Dana-Farber Cancer Institute in collaboration with Replimune Inc., who is providing the drug and funding for the study. [Extracted from the article]

A preprint abstract discusses the scheduling of oncolytic virus (OV) therapy in hepatocellular carcinoma (HCC) patients. The study compares the immunological response and clinical outcomes of single versus repeated doses of reovirus in cancer patients. The results indicate that a single dose of reovirus is as effective or better than repeated doses in inducing an anti-tumor immune response. Combination therapy with anti-PD-L1 shows potential in maximizing the benefits of a single dose while avoiding toxicities in the background liver. However, it is important to note that this preprint has not been peer-reviewed. [Extracted from the article]

New York University has been issued a patent for the induction and enhancement of antitumor immunity using Sindbis virus vectors that express immune checkpoint proteins. The invention aims to develop new therapies to enhance anticancer treatment and elicit an immune response against tumor and cancer cells. The patent describes the use of Alphavirus vectors, such as Sindbis virus, to encode immune checkpoint proteins or fragments thereof, which can be administered to subjects with cancer or tumors. The therapeutic composition includes a fusion polypeptide that combines an immune checkpoint protein with an immunoglobulin hinge region and an immunoglobulin heavy chain constant domain. [Extracted from the article]

Researchers from Arbutus Biopharma Corporation have developed a patent application for a new lipid formulation called serum-stable nucleic acid-lipid particles (SNALP) that can deliver therapeutic agents for the treatment of cancer. These lipid particles have shown increased potency and tolerability compared to other nucleic acid-lipid particle compositions. The formulations consist of nucleic acids, cationic lipids, non-cationic lipids, and conjugated lipids that prevent particle aggregation. This patent application describes the novel lipid formulation and its potential for safe and effective delivery of therapeutic agents for cancer treatment. [Extracted from the article]

A patent has been issued for a liposomal formulation of p-ethoxy nucleic acids, which are antisense oligonucleotides used to inhibit the expression of specific genes. The patent addresses the challenges of using these oligonucleotides in gene therapy, such as instability and low uptake. The invention incorporates p-ethoxy backbone linkages to improve stability and cellular uptake. The composition can be used to treat diseases like cancer, autoimmune diseases, and infectious diseases. [Extracted from the article]

A patent has been issued for incoherent field sonodynamic therapy, a method of treating cancer using ultrasound energy to activate a drug or sonosensitizer that targets cancer cells. The sonosensitizer produces reactive oxygen species when exposed to ultrasonic energy, which kills the cancer cells. The therapy system delivers a signal that reduces the amount of energy needed to destroy cancer cells, minimizing damage to healthy cells. The therapy can be used to treat tumors in different parts of the body and can enhance the effectiveness of other cancer treatments. The patent also includes details about the ultrasound transducer system used in the therapy, including specific sonosensitizers and frequency ranges for the energy profile. [Extracted from the article]

A recent study conducted at Babasaheb Bhimrao Ambedkar University in Lucknow, India, has developed a potential therapeutic strategy for treating HER2-positive metastatic breast cancer. The researchers created liposomes coated with TPGS-COOH and loaded with the anticancer drug Gefitinib and the natural compound Lycorine hydrochloride. These liposomes were then surface-functionalized with Trastuzumab (Trast) and the tumor-homing peptide iRGD. The study found that these dual-ligand liposomes showed enhanced cellular uptake and reduced cell migration in breast cancer cell lines. The researchers concluded that these liposomes could be a promising treatment option for HER2-positive metastatic breast cancer. [Extracted from the article]

A recent report from the University of Hertfordshire discusses the challenges faced in cancer therapy, such as toxic effects, non-selective targeting, and resistance to chemotherapy and radiotherapy. The report focuses on the potential of miRNA-34 (miR-34) as a promising candidate for cancer therapy due to its ability to inhibit tumorigenesis. However, there are challenges in the therapeutic application of miR-34, including degradation and immunoreactive adverse reactions. The report highlights the use of nanoparticles to address these challenges and enhance the therapeutic activity and targeting precision of miR-34. Despite advancements in cancer therapies, challenges in cancer treatment persist. [Extracted from the article]

Researchers from Osaka University have published a report on a novel therapeutic medicine for neuroendocrine cancer. The study focuses on RNA splicing regulation and its potential in treating aggressive neuroendocrine cancers such as small cell lung cancer and neuroendocrine prostate cancer. The researchers investigated the use of amido-bridged nucleic acid-based splice-switching oligonucleotides to alter the splicing of the RE1-silencing transcription factor, leading to significant antitumor effects in tumor xenograft models. This new therapeutic approach shows promise for treating intractable neuroendocrine cancers. [Extracted from the article]

A recent study conducted by researchers at China Pharmaceutical University explored the use of liposomal formulation to enhance the antitumor efficacy of cytarabine and idarubicin in the treatment of acute myeloid leukemia (AML). The researchers found that dual-loaded liposomes containing cytarabine and idarubicin showed enhanced antitumor efficacy. The liposomes had a size distribution of approximately 100 nm, high encapsulation efficiency, and in vitro stability for 21 days. The study suggests that this liposomal formulation could be a promising approach for improving the treatment of AML. [Extracted from the article]

A recent study conducted at Central University Rajasthan in India explores the challenges and opportunities in intravesical drug delivery approaches for the treatment of lower urinary diseases such as bladder cancer, interstitial cystitis, and urinary inconsistency. The unique anatomical structure of the bladder wall poses a challenge for drug delivery, but intravesical drug delivery systems (IVDDs) have been developed to achieve localized delivery with fewer adverse effects. Integrating nano-carriers, such as nanoparticles, liposomes, and polymeric micelles, with IVDDs can enhance drug penetration through the bladder's mucosal barrier and improve drug distribution within the bladder wall. The study also discusses recent advancements, clinical trials, and patents related to IVDDs for the treatment of lower urinary diseases. [Extracted from the article]

A recent report published by researchers from Kunming University of Science and Technology in China discusses the use of green carbon dots (CDs) in cancer gene therapy. Green CDs, derived from natural sources like fruits and vegetables, offer advantages such as cost-effectiveness, stability, simplicity, safety, and environmental friendliness. The report explores the synthesis methods and renewable sources of green CDs, as well as their optical features and various biological applications, including bioimaging, biosensing, drug/gene delivery, antimicrobial and antiviral effects, cancer diagnosis, and pharmaceutical formulations. The research highlights the versatility and potential of green CDs in biomedical and biotechnological applications. [Extracted from the article]

A report from Nantong University in the People's Republic of China discusses the use of lipid nanoparticles for precise cancer therapy. The researchers synthesized lipid nanoparticles, liposomes, and polymeric micelles with different compositions and surface charges, and compared their biodistribution in the body. They found that lipid nanoparticles showed improved biodistribution in the liver, spleen, and lung compared to liposomes and polymeric micelles. The researchers concluded that lipid nanoparticles have therapeutic superiority as carriers of chemotherapeutic agents and have potential for precise cancer therapy. [Extracted from the article]

A recent study from the University of Lodz in Poland explores the use of nanotechnology in bladder cancer therapy. The researchers highlight the limitations of traditional therapies and the potential benefits of nanomedicine, which involves using organic and inorganic molecules as carriers of active substances. The study discusses various types of nanoparticles, targeting mechanisms, and approved nanotherapeutics, as well as their applications in phototherapy, gene therapy, and immunotherapy. The researchers also summarize the current perspectives, advantages, and challenges in clinical translation. [Extracted from the article]

A recent report discusses advances in cancer gene therapy, focusing on the use of graphene quantum dots (GQDs) for drug and gene delivery in cancer treatment. GQDs have unique properties that make them promising agents for targeted and efficient treatment options. They can damage cancer cells and enhance their effectiveness through photothermal conversion and singlet oxygen generation. However, challenges such as achieving uniform size and morphology, bandgap engineering, and minimizing cytotoxicity and environmental impact need to be addressed. Integrating GQDs into existing drug delivery systems can improve the efficacy of traditional treatments and offer less invasive options for cancer patients. [Extracted from the article]

Researchers at the Institute of Oncology Ljubljana have conducted a non-clinical evaluation of pmIL12 gene therapy for the approval of a phase I clinical study. The therapy involves the use of interleukin-12 (IL-12), a cytokine known for its ability to mediate antitumor activity. The evaluation demonstrated the safety and efficacy of the therapy, with no detectable systemic toxicity. Approval has been obtained for the initiation of a phase I clinical study in basal cell carcinoma (BCC). [Extracted from the article]

A recent report from China Pharmaceutical University discusses the role of circular non-coding RNAs (circRNAs) in cancer progression and their potential impact on the effectiveness of immune checkpoint blockade (ICB) therapy in melanoma. The researchers identified a specific circRNA, circPIAS1, which is downregulated after coadministration of a PD-1 inhibitor. They found that circPIAS1 encodes a novel polypeptide, circPIAS1-108aa, which promotes cancer cell proliferation and inhibits immunogenic ferroptosis induced by ICB therapy. By modulating the balance between SUMOylation and phosphorylation of STAT1, circPIAS1-108aa hinders the effectiveness of ICB therapy. The study suggests that targeting circPIAS1 alongside PD-1 inhibitors may enhance the efficacy of immune drugs in melanoma treatment. [Extracted from the article]

A recent study conducted at the University of Missouri focused on the development of cancer gene therapy using luciferase bioluminescence. The researchers aimed to create an oncolytic adenovirus-resistant human triple-negative breast cancer cell line that could express luciferase. By combining this cell line with chemotherapies or targeted therapies, they were able to monitor the effectiveness of these compounds in real-time using bioluminescence imaging. The study found that the integration of luciferase affected the cells' response to different treatments, indicating the need for proper controls in future experiments. [Extracted from the article]

A recent study conducted by researchers at the University of Padua explores the potential of oncolytic viruses (OVs) as a new therapeutic tool for glioblastoma (GBM), the most aggressive brain tumor in adults. The study focuses on an oncolytic herpes simplex virus type 1 (oHSV-1) that has been modified to target and kill GBM cells. The results show that the oHSV-1 treatment significantly prolongs survival in mice with GBM tumors and stimulates an adaptive immune response, leading to the rejection of tumor cells. These findings suggest that this strategy could be applied in a clinical setting. [Extracted from the article]

Researchers at Case Western Reserve University have developed a customizable platform for cancer gene therapy that integrates chimeric antigen receptor (CAR) technology with the conditional expression of immunotherapeutics in T cells. The platform aims to overcome the challenges posed by immunosuppressive tumor microenvironments by combining CAR-T cells with therapeutic gene circuits that can sense cancer-associated signals and activate the production of customized therapeutic gene products. The researchers demonstrated that the engineered T cells could respond to recombinant b-galactosidase, leading to the production and secretion of various immunotherapeutics. This modular design has the potential to enhance CAR-T efficacy and persistence by locally producing antitumor therapeutics. [Extracted from the article]

Researchers from the University of Zurich have conducted a study on the variability of viral infections, specifically focusing on adenovirus infections in human lung epithelial cells. The study found that the variability in infection primarily arises from preexisting cell states rather than stochastic noise. The researchers discovered that certain cell states confer high or low susceptibility to adenovirus infection, indicating that the underlying cell state plays a crucial role in determining infection outcomes. These findings have implications for antiviral strategies and gene therapy applications. [Extracted from the article]

A study conducted by researchers at Zhejiang University in Hangzhou, China, has explored the use of nanovesicles in cancer gene therapy to prevent breast cancer liver metastasis. The researchers developed a nanovesicle that targeted activated hepatic stellate cells (aHSCs) and delivered a combination of drugs to inhibit fibrosis and restore the liver microenvironment. This approach successfully regressed aHSCs, restored immune function, and inhibited breast cancer hepatic metastasis in mouse models. The study highlights the potential of this nanomaterial vector for restraining breast cancer metastasis by targeting aHSCs, natural killer cells, and disseminated tumor cells. [Extracted from the article]

Researchers from the Graduate University of Advanced Technology in Kerman, Iran have developed a magnetic chitosan biopolymer that can be used as a non-virus vector for targeted gene delivery in cancer therapy. The nanocarrier was successfully synthesized and characterized, showing high gene expression and transfection without toxicity. The functionalized chitosan has excellent DNA condensation, protection, and high target transfection efficiency. This study suggests that the magnetic chitosan nanocarrier has potential applications in cancer therapy. [Extracted from the article]

A recent study conducted at Suez Canal University in Ismailia, Egypt, explores the potential use of nanodiamonds (NDs) in dentistry for therapeutic and regenerative applications. NDs have unique qualities such as nanometer size, target cell selectivity, and fluorescence, making them effective for tissue regeneration, gene therapy, and medication delivery. However, there is a lack of published research on the biological compatibility of NDs in dentistry, necessitating further investigation. The researchers believe that in the next decade, NDs will find various daily uses in dental practice. [Extracted from the article]

Tagworks Pharmaceuticals BV has appointed Ken Mills as its Chief Executive Officer and member of the Board of Directors. Mills, who previously served as CEO and President of REGENXBIO, brings valuable experience in bringing novel treatments to patients and company building. Tagworks is a precision oncology company that aims to develop a new standard of care for patients with solid tumors by targeting clinically validated tumor markers. Mills will work alongside Marc Robillard, the founding CEO of Tagworks, who will continue as Chief Scientific Officer and board member. The company has recently secured $65 million in series A financing and expects its first programs to enter the clinic in 2025. [Extracted from the article]

Researchers from the Fred Hutchinson Cancer Research Center in Seattle, Washington have conducted a phase 1 clinical trial on a novel T-cell therapy for recurrent leukemia after hematopoietic stem cell transplantation (HCT). The therapy involves genetically engineering T cells to express T cell receptors (TCR) specific for minor histocompatibility antigens. The study found that the therapy was feasible, safe, and showed preliminary signs of efficacy, with four patients achieving or maintaining complete remissions. The research provides valuable insights into the potential of T-cell therapy for treating leukemia after HCT. [Extracted from the article]

A recent study conducted by a researcher at Georgia State University has found that the combination of oncolytic virotherapy and immunotherapy has the potential to improve the treatment outcomes for triple-negative breast cancer (TNBC). The study focused on a Smac-armed oncolytic virus called VSV-S, which was shown to be more effective than wild-type VSV in treating TNBC in mouse models. The treatment was found to alter the tumor microenvironment, reducing immunosuppressive cells and increasing the infiltration of immune cells. The researchers also observed that neoadjuvant VSV-S treatment significantly reduced lung metastasis and increased survival rates in mice. The study suggests that this combination therapy could be a promising approach for TNBC treatment. [Extracted from the article]

Researchers at the University of Pennsylvania have discovered that small extracellular vesicles (sEVs) secreted by tumor cells act as a "forcefield," blocking the delivery of therapeutics to solid tumors. The team used gene-editing technology to halt the secretion of sEVs and found that this allowed nanoparticles loaded with therapeutics to penetrate tumor tissue more effectively. The findings open up possibilities for improving the delivery of nanoparticle-based treatments to solid tumors and developing strategies to disable the tumor's defense system. Further research will explore disrupting this defense system in different types of tumors. [Extracted from the article]

A study conducted by researchers at Chiba University explores the use of lipid nanoparticles (LNPs) for delivering small interfering RNA (siRNA) molecules to target cells. The researchers used NMR-based molecular-level characterization to investigate how different siRNA mixing methods affect the distribution and molecular state of siRNA within LNPs. They compared three preparation methods and found that pre-mixed LNPs exhibited a more uniform distribution of siRNA and superior gene-silencing effects compared to post-mixed LNPs. These findings have the potential to enhance gene therapies, RNA-based medicines, and RNA vaccines, leading to more effective and safer treatments for diseases like cancer and viral infections. Additionally, these advancements could contribute to the development of personalized medicine and increase access to innovative therapies. [Extracted from the article]

A patent has been issued to Ginkgo Bioworks Inc. for antibody-evading virus vectors. The patent describes methods and compositions involving adeno-associated virus (AAV) capsid proteins that have been modified to evade host antibodies. These modified AAV vectors have the potential to expand the eligible patient population for AAV-based gene therapy and allow for multiple administrations of AAV vectors. The patent also includes claims for recombinant AAV capsid proteins, AAV vectors, methods of introducing nucleic acid molecules into cells using AAV vectors, pharmaceutical compositions, and more. This patent provides valuable information for researchers and scientists working in the fields of biotechnology, gene therapy, and virology. [Extracted from the article]

A patent application titled "Personalized Vaccine Administration" has been published online by the US Patent and Trademark Office. The inventors propose a method of manufacturing and administering personalized vaccines to individuals. The method involves synthesizing a vaccine containing DNA, RNA, or peptides and packaging it in a cartridge that can be loaded into an injector. The personalized vaccine can be used to treat or ameliorate diseases related to mutations in individuals, such as tumor cancer. The inventors also describe an injector device that can be used for administering the personalized vaccine. This patent application highlights the potential for personalized approaches to vaccination and the use of non-viral gene delivery systems. [Extracted from the article]

Non-muscle-invasive bladder cancer (NMIBC) is a type of bladder cancer that remains confined to the innermost lining layer of the bladder without spreading into the muscle. It is a common form of bladder cancer and is usually associated with superficial tumor growth. The current treatment plan for NMIBC involves surgery, intravesical immunotherapy, and intravesical chemotherapy. The FDA has approved three drugs for treating NMIBC: KEYTRUDA, ADSTILADRIN, and ANKTIVA. There are also several other potential therapies in development for NMIBC. The market size for NMIBC is expected to grow significantly in the coming years. [Extracted from the article]

This article discusses a clinical trial, NCT06609928, that is testing the safety and effectiveness of FH-FOLR1 CAR T-cell therapy in treating pediatric patients with relapsed or refractory AML. The trial involves modifying a patient's T cells in the lab to attack cancer cells. The study aims to determine if this therapy is safe and tolerable for pediatric patients with recurrent or refractory AML. The trial involves apheresis, chemotherapy, and infusion of FH-FOLR1 CAR T cells, with a 15-year follow-up. Additionally, the document provides alternative names for medical procedures and drugs, which can be helpful for library patrons conducting research on these topics. [Extracted from the article]

A patent application titled "Mesenchymal Stem Cell-Derived Exosome Drug Delivery For Cancer And Other Disorders" has been filed by inventor Carl Randall Harrell. The application discusses the potential of mesenchymal stem cells (MSCs) and exosomes derived from MSCs (MSC-Exos) in treating various diseases, including cancer. MSC-Exos are nano-sized extracellular vesicles that can deliver bioactive substances, such as chemotherapeutics, directly into tumor cells. The application also mentions the use of peptides to improve the targeting of MSC-Exos in cancer-related applications. The invention includes methods and compositions for producing and using exosomes as treatments or drug delivery systems. [Extracted from the article]

A patent has been issued to Houston Gene Therapeutics LLC for compositions that can be used to treat vascular diseases. The patent describes the use of adeno-associated virus (AAV) vectors to deliver therapeutic genes for the treatment and prevention of multifactorial disorders such as heart disease, inflammation, cancer, dementia, obesity, and type 2 diabetes. The patent also discusses the development of small regulated promoters that can control the expression of therapeutic genes in response to specific stimuli. The invention aims to address the need for effective therapies for high-lipid associated disorders, such as atherosclerosis, by developing animal models and therapeutic methods. [Extracted from the article]

A clinical trial, NCT06613152, is currently underway to assess the safety and effectiveness of HS-IT201 injection in treating advanced solid tumors. The trial involves adoptive cell therapy using autologous tumor-infiltrating lymphocytes (TIL) after lymphodepletion preparative treatment. The study is in its early stages and is not yet recruiting participants. The trial will measure adverse events, objective response rate, time-to-response, duration of response, disease control rate, progression-free survival, overall survival, lymphocyte subpopulation, and TCR repertoire. The study is open to individuals of all genders between the ages of 18 and 75, and contact information for the study is provided. The trial is being conducted by Qingdao Sino-Cell Biomedicine Co., Ltd. [Extracted from the article]

A patent has been issued to Cg Oncology Inc. for methods of treating solid or lymphatic tumors using combination therapy. The patent describes a method that involves locally administering an oncolytic virus to the tumor site and systemically administering an immunomodulator. The oncolytic virus is designed to replicate in cancer cells and contains a gene encoding an immune-related molecule. The immunomodulator helps activate the immune system to fight against the tumor. This method aims to improve the efficacy of cancer immunotherapy, which has faced challenges in the past. [Extracted from the article]

A patent application titled "Armed Seneca Valley Virus Oncolytic Therapy Compositions And Methods Thereof" has been published online. The application discusses the use of the Seneca Valley virus (SVV) as a potential anti-cancer agent. The inventors propose genetically engineering the SVV to express therapeutic proteins that can be used to treat various types of cancer. The application also mentions the use of armed SVV in combination with other cancer treatments to enhance their effectiveness. This patent application describes a promising approach to cancer treatment using armed SVV. [Extracted from the article]

A patent application titled "Methods And Compositions For Sensitization Of Tumor Cells To Immune Therapy" has been published online. The inventors propose methods and compositions for increasing the sensitivity of cancer cells to T cell killing, specifically through the inhibition of autophagy and/or the NF-kB pathway. These methods aim to address the challenge of tumor cell resistance to immunotherapies. The patent application also mentions the potential use of additional anti-cancer therapies, such as cancer immunotherapy, in combination with the proposed methods. [Extracted from the article]

A preprint abstract from biorxiv.org discusses a strategy for using retroviral vectors to deliver genes to tumor-specific T cells in mice. The study aims to improve the efficacy of TIL therapy, which harnesses endogenous T cells to fight tumors. By pseudotyping retroviral vectors with peptide-major histocompatibility complexes (pMHC), the researchers were able to specifically deliver function-enhancing cargoes to CD8 T cells and expand anti-tumor T cells in vivo. This approach has the potential to streamline engineered cell therapy production for various applications. However, it is important to note that this preprint has not yet undergone peer review. [Extracted from the article]

A preprint abstract discusses the challenges of distributing oncolytic viruses (OVs) within tumors and the lack of predictive biomarkers for treatment outcomes. The study used multimodal bioluminescence imaging and magnetic resonance imaging to monitor OV infection, replication, and tumor viability in brain glioma mouse models. The researchers found that microglia and tumor necrosis hindered OV distribution within the tumor, and they incorporated a CSF-1 receptor inhibitor (PLX3397) to improve tumor control by reducing microglial interference. The findings suggest that targeting brain-resident immune cells could enhance the efficacy of OVs in resistant brain tumors. [Extracted from the article]

A preprint abstract discusses the potential use of Zika virus (ZIKV) peptides in immunotherapy for brain tumors. The study found that ZIKV infects and kills aggressive cancer cells in brain tumors, and T cell recruitment into ZIKV-infected brain tumors is well-documented. The researchers used a combined LC-MS/MS global proteome and immunopeptidome approach to identify ZIKV peptides presented on the surface of infected brain tumor cells. They identified several ZIKV peptides that could potentially be used to develop novel immunotherapies for brain tumors. However, it is important to note that this preprint has not been peer-reviewed. [Extracted from the article]

A patent has been issued for compositions and methods related to megakaryocyte-derived extracellular vesicles. These vesicles have unique biomarker and size profiles that make them suitable for therapeutic delivery and treating various diseases. They can be used for drug delivery and treatment of genetic disorders, infectious diseases, hematopoietic diseases, and hemoglobinopathies. The patent describes the composition of the vesicles, their ability to home to bone marrow cells, and their potential for loading with therapeutic agents. The invention aims to provide cost-effective and safe delivery vehicles for therapeutic agents. [Extracted from the article]

Biogen MA Inc. has been issued a patent for compositions that can modulate the expression of tau mRNA and protein. Tau is a protein associated with various brain diseases, including Alzheimer's disease and frontotemporal dementia. The patent describes methods, compounds, and compositions for reducing tau levels, which could potentially be used to prevent, treat, or ameliorate neurodegenerative diseases. The patent also includes claims for specific compounds and compositions, as well as methods of treatment. This information could be valuable for researchers and individuals interested in understanding and developing treatments for tau-associated disorders. [Extracted from the article]

A patent application has been submitted for a method of modulating the expression of the ACMSD protein in target cells. The application describes an expression construct that can deliver the ACMSD protein to various target cells, including those in the central nervous system. This method could potentially be used to treat diseases such as Parkinson's disease, Alzheimer's disease, ALS, and mood disorders. The application provides detailed information about the constructs, vectors, and methods involved in this system. [Extracted from the article]

The patent application titled "INVERTED CHIMERIC siRNA MOLECULES AND METHODS OF USE THEREOF" describes the invention of siRNA molecules that can inhibit the expression of specific genes, including c-Myc and KRAS, simultaneously. These molecules are more effective than single siRNAs and serial chimera siRNAs. The application also provides information on the composition and pharmaceutical composition of these siRNA molecules, as well as methods for using them to inhibit gene expression and treat cancer. [Extracted from the article]

The patent application titled "Targeted Rna Editing By Leveraging Endogenous Adar Using Engineered Rnas" discusses methods for editing target RNA in host cells using engineered RNA molecules. These methods involve introducing a construct containing a deaminase-recruiting RNA (dRNA) into the host cell, which can recruit an adenosine deaminase acting on RNA (ADAR) to edit the target RNA. The application also mentions the limitations of current ADAR-mediated RNA editing technologies and proposes alternative approaches to overcome these limitations. The methods described in the patent application can be used to edit various types of RNA associated with diseases such as cancer, Mucopolysaccharidosis type I, Ehlers-Danlos syndrome, Joubert syndrome, Fanconi anemia, primary familial hypertrophic cardiomyopathy, and X-linked severe combined immunodeficiency. Detailed information on the specific techniques and embodiments involved in the methods is provided in the application. [Extracted from the article]

A patent application has been submitted for a new approach to cancer gene therapy. The application describes the use of genetically engineered immune cells, called CAR-T cells, that have the potential to recognize and destroy cancer cells more effectively. However, there are still limitations to this therapy, such as off-target toxicity and reduced efficacy. The inventors propose using a combination of priming receptors and CARs to address these challenges. The patent application provides detailed methods and compositions for treating diseases, particularly cancer, using immune cells that express specific nucleic acid sequences. [Extracted from the article]

The US Patent and Trademark Office has published a patent application titled "Tracking Apobec Mutational Signatures In Tumor Cells." The application, filed by inventors from New York, NY, US; Cambridge, MA, US; and London, GB, and assigned to Broad Institute Inc., outlines a method for identifying and quantifying the mutagenic impact of a specific pathway in tumor cells. The invention aims to track tumor mutagenesis over time to guide treatment, inform cancer diagnosis, and prognosis. The application also discusses the role of APOBEC3 deaminases in cancer mutagenesis and potential therapeutic interventions targeting these enzymes. Methods for inhibiting APOBEC deaminases and DNA repair proteins as a potential treatment for various types of cancer are described, as well as methods for identifying patients likely to respond to inhibitors of these proteins. Additionally, the application presents a method for tracking mutagenesis induced by a specific gene in a population of cells over time. [Extracted from the article]

A patent application by inventor Michael Heartlein has been made available online for a new method of gene therapy. The invention aims to address the limitations and risks associated with conventional gene therapy methods by using non-immunogenic components and leveraging endogenous human gene processing machinery. The method involves delivering a gene transfer construct comprising a reverse complement sequence for a protein of interest coupled with L1 retro elements. The invention combines the efficiency of mRNA transcript therapies with the durability of gene therapy and gene editing technologies. The composition can be encapsulated in a nanoparticle delivery vehicle for administration. [Extracted from the article]