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Researchers from Kangwon National University in South Korea have published a report on the use of nanoparticles as non-viral vectors for the delivery of CRISPR/Cas9 gene-editing technology. The study explores the efficiency and limitations of various types of nanoparticles, including lipid-based, polymer-based, inorganic, and extracellular vesicle-based systems. The researchers aim to provide a comprehensive overview of the role of non-viral delivery systems in advancing CRISPR/Cas9 technology for clinical applications and gene therapy. This research is supported by various organizations, including the National Research Foundation of Korea and the Ministry of Science and ICT. [Extracted from the article]

A recent report from the Spanish National Research Council (CSIC) provides new insights into gene therapy. The researchers studied the crystal structure of six cationic cholesteric liquid crystal polyesters with choline, ammonium, and amide end groups. These polyesters have demonstrated non-viral vector properties in gene therapy by delivering DNA to the cell nucleus. The researchers also observed unexpected optical activity and chiral morphology in the macromolecular structures of these polyesters. The morphology of the polyesters was further studied using ESEM. This research sheds light on the crystal structure and properties of these cationic polymers, which could have implications for gene therapy applications. [Extracted from the article]

A recent study conducted by researchers at Pontifical University Javeriana has explored the potential of a CRISPR-nCas9-based gene therapy strategy for treating Mucopolysaccharidosis (MPS) IVA, a lysosomal storage disorder. The researchers used iron oxide nanoparticles as non-viral vectors to deliver the human GALNS cDNA into the ROSA26 locus in an MPS IVA mouse model. The results showed increased GALNS activity, reduction of keratan sulfate, partial recovery of bone pathology, and no toxicity or immune response activation. This study provides in vivo evidence of the potential of CRISPR-nCas9-based gene therapy for treating MPS IVA using non-viral vectors. [Extracted from the article]

A recent study from Amirkabir University of Technology explores the use of carbon-based nanostructures as emerging materials for gene delivery applications in gene therapy. The researchers highlight the potential of nanostructures, such as carbon nanotubes and nanodiamonds, as non-viral vectors for delivering genetic material. They discuss the advantages and challenges of using carbon-based nanostructures in gene therapy, emphasizing the need for advancements in their features to improve efficiency and reduce toxicity. The study provides insights into the synthesis methods, properties, and biomedical applications of carbon-based nanostructures in gene delivery systems. [Extracted from the article]

Federal University Rio de Janeiro, Biotechnology, Viral, Virus, Genetics, Gene Therapy, Bioengineering, Nanotechnology, Drugs and Therapies, Emerging Technologies, Therapy, Nanosystems Keywords: Federal University Rio de Janeiro; Biotechnology; Viral; Virus; Genetics; Nanosystems; Gene Therapy; Bioengineering; Nanotechnology; Drugs and Therapies; Emerging Technologies; Therapy EN Federal University Rio de Janeiro Biotechnology Viral Virus Genetics Nanosystems Gene Therapy Bioengineering Nanotechnology Drugs and Therapies Emerging Technologies Therapy Fresh data on gene therapy are presented in a new report. [Extracted from the article]

Researchers from the All India Institute of Medical Sciences (AIIMS) have conducted studies on gene therapy for hearing loss, focusing on genetic factors behind human hearing impairment. The review article discusses screening approaches and next-generation sequencing technologies to diagnose patients with hearing impairments accurately. The research emphasizes the potential of gene therapy techniques for restoring or preserving hearing, using viral or non-viral vectors to deliver genes to target cells. This study provides valuable insights into the development of gene therapy for hearing loss. [Extracted from the article]

Researchers from the California Institute of Technology have conducted a study on adeno-associated viruses (AAVs), which are commonly used in gene therapy. The researchers used a human cell microarray platform to analyze the interactions between AAVs and human cells. They discovered specific interactions between the AAV9 capsid and human interleukin 3 (IL3), as well as interactions with low-density lipoprotein receptor-related protein 6 (LRP6) that enhance the AAVs' ability to cross the blood-brain barrier. These findings provide valuable insights for the development of improved viral and non-viral vectors for therapeutic delivery to the brain. [Extracted from the article]

A recent preprint abstract discusses the use of adeno-associated viruses (AAVs) in gene therapy. The study utilized a human cell microarray platform to examine the interactions between natural and engineered AAVs and human cells. The researchers identified specific interactions between the AAV9 capsid and human interleukin 3 (IL3), as well as interactions with low-density-lipoprotein-receptor-related-protein 6 (LRP6) that may allow engineered AAVs to cross the blood-brain barrier. The findings provide valuable insights for the development of improved viral and non-viral vectors for therapeutic delivery to the brain. However, it is important to note that this preprint has not yet undergone peer review. [Extracted from the article]

A recent study from Tsinghua University in Beijing, China, explores the challenges and potential of mRNA-based delivery of gene editing agents in gene therapy. The researchers compare different gene editing agents and discuss various delivery systems, including viral and non-viral vectors. They also highlight the importance of the protein corona in nanodelivery. The purpose of this review is to guide further research and provide a comprehensive analysis of mRNA-based in vivo delivery of gene editing agents. [Extracted from the article]

A recent study conducted by researchers at Nagasaki University in Japan explores the use of non-viral vectors in gene therapy. The study focuses on the interaction between serum components and gene transfer using polyethyleneimine (PEI) and g-polyglutamic acid (g-PGA) complexes. The researchers found that heat-labile components in the serum contribute to gene expression in binary complexes, while serum albumin and salts affect gene expression in ternary complexes. The study suggests that changes in particle size and z-potential may play a role in enhancing gene expression. These findings provide valuable insights into the use of polymers like PEI and g-PGA in non-viral gene transfer. [Extracted from the article]

A recent report from the Academy of Sciences of the Czech Republic discusses the potential of siRNA-based therapy and the challenges in delivering siRNA effectively. The researchers have developed non-viral vectors based on modified nanodiamonds that show promise in siRNA delivery. By optimizing variables such as colloidal stability, surface properties, and polymer composition, they have created stable siRNA delivery tools with potential for RNA interference-based therapeutics. The study presents a novel copolymer-coated nanodiamond vector that demonstrates potential for siRNA transfection and RNA interference-based therapeutics. [Extracted from the article]

A patent has been issued to Abelzeta Inc. for a method of large-scale preparation of purified recombinant lentiviral vectors at GMP grade. Gene therapy involves introducing therapeutic genes into target cells to treat diseases caused by gene defects. Lentiviral vectors are commonly used in gene therapy due to their ability to infect both dividing and non-dividing cells. The current method of obtaining lentivirus through ultracentrifugation is not suitable for large-scale production, and existing chromatography purification methods have limitations. This patent presents a new method for preparing purified lentivirus vectors that is efficient, suitable for large-scale production, and meets the requirements of GMP grade production. [Extracted from the article]

A patent application has been submitted for "Modified Polypeptides And Uses Thereof" to the US Patent and Trademark Office. The application focuses on the role of cyclin F polypeptides in treating neurodegenerative diseases like ALS and FTD. The researchers have found that targeting cyclin F polypeptides to the cytoplasm of cells can effectively bind and clear cytoplasmic TDP-43, a protein associated with ALS and FTD, while leaving the nuclear form of TDP-43 untouched. This discovery has potential implications for enhancing neuron survival and treating neurodegenerative conditions. The application describes modified cyclin F polypeptides and their potential uses in enhancing neuron survival, inhibiting degeneration and abnormal protein accumulation in neurons, and treating neurodegenerative conditions. The modified polypeptides contain a heterologous nuclear export signal (NES) and/or a deletion of the PEST domain. They may also have a Nuclear Localization Signal (NLS)-inactivating modification. The polypeptides can bind to TDP-43 and retain certain functional abilities of the wild-type cyclin F polypeptide. The application also mentions delivery vehicles, such as viral vectors or non-viral vectors, for delivering the nucleic acid molecules encoding the modified polypeptides. [Extracted from the article]

Acrylates, Acyclic Acids, Bioengineering, Biotechnology, CRISPR, Drugs and Therapies, Gene Editing, Gene Therapy, Genetics, Genome Editing, University of Western Australia Keywords: Acrylates; Acyclic Acids; Bioengineering; Biotechnology; CRISPR; Drugs and Therapies; Gene Editing; Gene Therapy; Genetics; Genome Editing; University of Western Australia EN Acrylates Acyclic Acids Bioengineering Biotechnology CRISPR Drugs and Therapies Gene Editing Gene Therapy Genetics Genome Editing University of Western Australia 3789 3789 1 10/09/23 20231009 NES 231009 2023 OCT 13 (NewsRx) -- By a News Reporter-Staff News Editor at Gene Therapy Weekly -- University of Western Australia (Crawley, Australia) has been issued patent number 11760823, according to news reporting originating out of Alexandria, Virginia, by NewsRx editors. "Liposomal and viral vectors have been used for the concurrent delivery of multiple components (for example, CRISPR and Cas9 plasmid, together with multiple guide RNAs) for effective or CRISPR-based genome editing. [Extracted from the article]

Recent advancements in ocular drug delivery systems, as reported by a study from a Military Hospital in Bareilly, India, focus on improving drug penetration, sustaining drug release, and enhancing patient compliance. These innovations include nanotechnology-based delivery systems, sustained-release implants, hydrogels, microneedle technology, and gene therapy vectors. The study highlights the potential of these novel delivery systems to provide controlled and sustained drug release, improve drug stability, and target specific ocular tissues, ultimately leading to enhanced therapeutic outcomes and reduced side effects. Researchers aim to address ongoing challenges and explore future research avenues in the field of ocular drug delivery. [Extracted from the article]

A patent application titled "Method For Large-Scale Preparation Of Purified Preparation Of Recombinant Lentiviral Vector At Gmp Grade" has been published online by Abelzeta Inc. The inventors have developed a method for purifying lentivirus vectors used in gene therapy. The method involves microfiltration, concentration, chromatography, and liquid exchange to obtain purified recombinant viral vectors. The method aims to meet the requirements of large-scale production and GMP grade production. The patent application provides detailed steps and preferred embodiments of the method, as well as a purification device for implementing the method. [Extracted from the article]

A patent application titled "Central Nervous System Targeting Polynucleotides" has been published online by the US Patent and Trademark Office. The inventors of the application propose the use of adeno-associated virus (AAV) to deliver therapeutic agents to the central nervous system (CNS) for the treatment of CNS disorders. They discuss the need for better understanding and optimization of delivery parameters for viral particle distribution to achieve safer and more effective gene therapy. The application also describes the design, preparation, and formulation of AAV particles, as well as methods for their administration and delivery for the treatment of CNS diseases or disorders. [Extracted from the article]

A recent report from researchers at Renmin Hospital of Wuhan University in China discusses the challenges and progress in gene therapy for retinitis pigmentosa (RP), a hereditary eye disease that affects the retina. RP is characterized by the loss of photoreceptor cells and atrophy of the retinal pigment epithelium, and finding an effective treatment has been difficult due to the genetic diversity of the disease. However, advancements in gene therapy, including gene-editing techniques and the discovery of new compounds and gene loci, offer hope for more effective treatments. The report focuses on the latest applications of gene editing research in RP, discussing the most prevalent genes associated with the disease and advancements in genome-editing strategies. [Extracted from the article]

Research from the University of Porto in Portugal has explored the use of cationic serine-based gemini surfactants as potential gene delivery agents. These surfactants can co-assemble with DNA due to strong electrostatic associations and can enhance DNA complexation when combined with a helper lipid. The study found that mixtures of serine-based gemini surfactants and monoolein as a helper lipid were non-cytotoxic and effectively complexed DNA. The research also observed that the addition of monoolein affected the morphology and size distribution of the formed aggregates. The findings suggest that lipoplexes, formed by these surfactants, may have a similar bilayer-based structure. [Extracted from the article]

Researchers at Zhengzhou University in China have conducted a review on nucleic acid nanodelivery systems for the treatment of inflammatory diseases. The study highlights the potential of nucleic acids as therapeutic drugs and carriers, which can intervene in diseases at the source. The researchers discuss different nucleic acid drugs and their delivery vehicles, emphasizing the advantages of substance-modified spherical nucleic acids and tetrahedral framework nucleic acids. The study concludes that innovation in nucleic acid nano-delivery systems is crucial for achieving a wider range of applications in disease therapy. This research has been peer-reviewed and provides valuable insights into the field of gene therapy. [Extracted from the article]

A recent study conducted by researchers at the Materials and Energy Research Center in Karaj, Iran, explores the use of metallic nanoparticles (MNPs) in gene therapy. MNPs have gained attention for their ability to enhance the effectiveness of medications by reducing multidrug resistance and delivering therapeutic agents directly to targeted tissues. In addition to drug delivery, MNPs have various medical applications, including diagnostics and improving the biocompatibility of materials. The study focuses on the preparation methods, characterization techniques, and theranostic applications of MNPs, as well as their challenges in clinical trial translation. For more information, readers can refer to the research article published in Frontiers in Chemistry. [Extracted from the article]

A new report discusses research findings on gene therapy, specifically focusing on potential targeting mechanisms for bone-directed therapies. The study highlights the complex regulation mechanisms involved in bone development and the challenges of drug delivery to the skeletal system. While current strategies such as recombinant enzymes, hormone infusions, gene delivery, and cell therapy have shown some improvement in bone lesions, there is a need for novel delivery methods that address target specificity, immunogenicity, and duration in circulation. The report concludes that targeted drug delivery using organic and inorganic compounds holds promise for future bone-targeting systems. [Extracted from the article]

A patent application has been submitted for a method of gene therapy called electroporation gene therapy for tissue barriers. The method involves introducing genetic treatment material near a tissue barrier and using electroporation to transfer the genetic material into the cells of the barrier. The patent application provides various methods and techniques for delivering the genetic material and performing electroporation. This research has potential applications in the field of medical devices and could contribute to the development of new therapies for various diseases and conditions. [Extracted from the article]

Researchers from the University of Tabriz have published a study on recent advances in gene therapy. Gene therapy is a treatment option for diseases that do not respond well to traditional medication. The study focuses on spherical nucleic acids (SNAs), which are nanotechnology-based gene delivery systems. SNAs have unique structural properties that make them effective in delivering genetic materials into cells, with high stability and minimal toxicity. The researchers discuss the types and optimization mechanisms of SNAs, as well as recent advancements in the synthesis of gene delivery nanoplatforms based on SNAs. [Extracted from the article]

The patent application by Sichuan Real&Best Biotech Co. Ltd. outlines a pharmaceutical composition for a recombinant adeno-associated virus (rAAV) vector used in gene therapy. The composition includes specific components such as salts, buffers, stabilizers, and surfactants. The rAAV vector has been proven effective in targeting genes in different tissues and the composition is designed to remain stable for over a year under refrigeration. The patent application provides detailed information on the components and concentrations used in the composition. For further details, please refer to the complete patent application. [Extracted from the article]

The US Patent and Trademark Office has published a patent application titled "Assay for Massive Parallel RNA Function Perturbation Profiling." The application, filed by Rabia Khan of Ladder Therapeutics Inc., outlines a method for studying the effects of chemical or genetic perturbations on RNA structures within cells. This method involves using nucleic acid constructs and vectors to investigate the functional nature of RNA structures and screen for candidate agents that can interact with these structures. The technology has the potential to advance drug discovery and target new biological processes. The patent application provides detailed information on the methodology and potential applications of this screening method, which involves identifying cell populations with altered gene expression after exposure to candidate agents. The RNA constructs used in the method may contain various structural elements such as G-quadruplexes, triple helices, pseudoknots, stem-loops, or multiway junctions. [Extracted from the article]

Researchers from Babes-Bolyai University in Cluj-Napoca, Romania have conducted a study on the interaction between hyperbranched polyethylenimine polyethylene glycol (HPEI-PEG) and DNA for gene delivery. Using molecular dynamics simulations, they investigated the effects of different numbers of PEG chains per HPEI core and developed a new CHARMM Force Field for PEG. The study found that the addition of PEG reduced cytotoxicity and increased solubility, while still maintaining high efficiency in forming polyplexes with DNA. The research provides insights into the dynamics of hydrogen bond formation between the HPEI core and DNA. This study was supported by Babes-Bolyai University and has been peer-reviewed. [Extracted from the article]

Gene therapy has made significant advancements in the treatment of genetic diseases, particularly rare and monogenic diseases. Clinical trials are currently being conducted, especially in the field of pediatric neurology, to evaluate the safety and efficacy of gene therapy. However, gene therapy still faces challenges such as cost, technical complexity, efficient gene delivery, immune response to vectors, and duration of therapeutic response. Advances in gene editing technology, such as CRISPR-Cas9, are expected to improve precision and efficiency in gene modification. Research on gene therapy vectors is also underway to enhance delivery capacity and safety of treatments. [Extracted from the article]

Researchers from Inha University in Incheon, South Korea, have published a perspective article exploring the use of carbon-based nanomaterials as alternatives to viral vectors for efficient CRISPR delivery in gene therapy. The article emphasizes the importance of design strategies in enhancing the interactions between CRISPR components and carbon-based nanomaterials. The researchers present specific case studies to demonstrate the effectiveness of carbon-based nanomaterials in CRISPR delivery and highlight the potential of these materials in the field of non-viral CRISPR delivery vectors. The insights provided contribute to the ongoing efforts to develop safe and efficient methods for gene delivery and therapy. [Extracted from the article]

In the present article, we review the most recent updates in clinical trials targeting the management of STGD1, including gene therapy, small molecule therapy, and stem cell therapy. Keywords: Bioengineering; Biotechnology; Clinical Research; Clinical Trials and Studies; Drugs and Therapies; Gene Therapy; Genetics; Health and Medicine; Macular Degeneration; Post-Trial Research; Stargardt Disease; Stem Cell Research; Therapy EN Bioengineering Biotechnology Clinical Research Clinical Trials and Studies Drugs and Therapies Gene Therapy Genetics Health and Medicine Macular Degeneration Post-Trial Research Stargardt Disease Stem Cell Research Therapy 1700 1700 1 10/16/23 20231017 NES 231017 2023 OCT 16 (NewsRx) -- By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on gene therapy have been presented. [Extracted from the article]

"The present use of the dyes such as vital dyes allows delivery of a therapeutic agent to the retina and/or choroid of an eye of a subject. The method according to claim 3, wherein the dye is a free dye, an aggregate of the dye, or a crystal of the dye; wherein the dye is conjugated to a further agent; wherein the dye is comprised in a nanoparticle; and/or wherein the dye and the therapeutic agent are comprised in a nanoparticle. Thereby, the present use of the dyes such as vital dyes allows treatment, such as laser-assisted treatment, of a retinal disease or choroidal disease. [Extracted from the article]

Keywords for this news article include: Center for Scientific Research, Biotechnology, Genetics, Gene Therapy, Nanoparticles, Bioengineering, Nanotechnology, Drugs and Therapies, miRNA-Based Therapy, Emerging Technologies. Keywords: Bioengineering; Biotechnology; Drugs and Therapies; Emerging Technologies; Gene Therapy; Genetics; Nanoparticles; Nanotechnology; miRNA-Based Therapy EN Bioengineering Biotechnology Drugs and Therapies Emerging Technologies Gene Therapy Genetics Nanoparticles Nanotechnology miRNA-Based Therapy 642 642 1 09/19/23 20230918 NES 230918 2023 SEP 22 (NewsRx) -- By a News Reporter-Staff News Editor at Gene Therapy Weekly -- New study results on miRNA-based therapy have been published. [Extracted from the article]

New siRNA-Based Therapy Study Findings Have Been Reported from University of L'Aquila (Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. For more information on this research see: Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Keywords: Bioengineering; Biotechnology; Bone Diseases and Conditions; Bone Research; Drugs and Therapies; Emerging Technologies; Gene Therapy; Genetics; Health and Medicine; Musculoskeletal Diseases and Conditions; Nanoparticles; Nanotechnology; Osteochondrodysplasias; Osteopetrosis; Osteosclerosis; Small Interference RNAs (siRNAs); Therapy; siRNA; siRNA-Based Therapy EN Bioengineering Biotechnology Bone Diseases and Conditions Bone Research Drugs and Therapies Emerging Technologies Gene Therapy Genetics Health and Medicine Musculoskeletal Diseases and Conditions Nanoparticles Nanotechnology Osteochondrodysplasias Osteopetrosis Osteosclerosis Small Interference RNAs (siRNAs) Therapy siRNA siRNA-Based Therapy 1290 1290 1 09/19/23 20230918 NES 230918 2023 SEP 22 (NewsRx) -- By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on siRNA-based therapy have been presented. [Extracted from the article]

Basal Ganglia Diseases and Conditions, Bioengineering, Biotechnology, Brain Diseases and Conditions, Central Nervous System Diseases and Conditions, Drugs and Therapies, Gene Therapy, Health and Medicine, Movement Disorders, Nervous System Diseases and Conditions, Neurodegenerative Diseases and Conditions, Parkinson's Disease, Parkinsonian Disorders, Risk and Prevention, Therapy Keywords: Basal Ganglia Diseases and Conditions; Bioengineering; Biotechnology; Brain Diseases and Conditions; Central Nervous System Diseases and Conditions; Drugs and Therapies; Gene Therapy; Health and Medicine; Movement Disorders; Nervous System Diseases and Conditions; Neurodegenerative Diseases and Conditions; Parkinson's Disease; Parkinsonian Disorders; Risk and Prevention; Therapy EN Basal Ganglia Diseases and Conditions Bioengineering Biotechnology Brain Diseases and Conditions Central Nervous System Diseases and Conditions Drugs and Therapies Gene Therapy Health and Medicine Movement Disorders Nervous System Diseases and Conditions Neurodegenerative Diseases and Conditions Parkinson's Disease Parkinsonian Disorders Risk and Prevention Therapy 2178 2178 1 08/14/23 20230815 NES 230815 2023 AUG 18 (NewsRx) -- By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on gene therapy. Keywords for this news article include: University of Brasilia, Brasilia, Brazil, South America, Biotechnology, Gene Therapy, Bioengineering, Movement Disorders, Drugs and Therapies, Health and Medicine, Parkinson's Disease, Risk and Prevention, Parkinsonian Disorders, Brain Diseases and Conditions, Basal Ganglia Diseases and Conditions, Neurodegenerative Diseases and Conditions. [Extracted from the article]

Barcelona, Spain, Europe, Bioengineering, Biotechnology, Drugs and Therapies, Gene Therapy, Genetics, Health and Medicine, Pharmacokinetics, Pharmacology Keywords: Barcelona; Spain; Europe; Bioengineering; Biotechnology; Drugs and Therapies; Gene Therapy; Genetics; Health and Medicine; Pharmacokinetics; Pharmacology EN Barcelona Spain Europe Bioengineering Biotechnology Drugs and Therapies Gene Therapy Genetics Health and Medicine Pharmacokinetics Pharmacology 1095 1095 1 08/07/23 20230808 NES 230808 2023 AUG 11 (NewsRx) -- By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators publish new report on Biotechnology - Gene Therapy. [Extracted from the article]

The method of claim 8, further comprising introducing a transgene comprising a fragment of the CFTR gene into the cell, such that upon integration of the transgene into the CFTR gene, a functional CFTR protein is expressed. In some embodiments, the transgene comprising a fragment of the CFTR gene such that upon integration of the transgene (e.g., into intron 1, 3, 7 or 8) into the CFTR gene, a functional CFTR protein is expressed in the cell. The primary defect in CF is in the regulation of epithelial chloride transport by a chloride channel protein encoded by the cystic fibrosis transmembrane conductance regulator (CFTR) gene. [Extracted from the article]