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191 results on '"Ciaramella, Giuseppe"'

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1. Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia

3. Improved cytosine base editors generated from TadA variants

4. Cytosine base editing enables quadruple-edited allogeneic CART cells for T-ALL

6. Base-editing corrects metabolic abnormalities in a humanized mouse model for glycogen storage disease type-Ia

7. A multiclade env–gag VLP mRNA vaccine elicits tier-2 HIV-1-neutralizing antibodies and reduces the risk of heterologous SHIV infection in macaques

8. In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

9. Installation of HbG-Makassar by base editing restores hemoglobin function: a transformative therapy for sickle cell disease

12. Cytosine base editing inhibits Hepatitis B Virus replication and reduces HBsAg expression in vitro and in vivo

14. Comprehensive genome editing confers ‘off-the-shelf’ CAR-T cells superior efficacy against solid tumors

16. Vaccine Mediated Protection Against Zika Virus-Induced Congenital Disease

19. Modified mRNA/lipid nanoparticle-based vaccines expressing respiratory syncytial virus F protein variants are immunogenic and protective in rodent models of RSV infection

21. High-efficiency base editing for Stargardt disease in mice, non-human primates, and human retina tissue

22. Development of adult hepatocyte culture systems capable of expressing genes coding for the major proteins responsible for foreign compound metabolism

23. Engineered Stem Cell Antibody Paired Evasion 1 (ESCAPE-1): Paired HSC Epitope Engineering and Upregulation of Fetal Hemoglobin for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for the Treatment of Hemoglobinopathies

24. Engineered Stem Cell Antibody Paired Evasion-2 (ESCAPE-2): Paired HSC Epitope Engineering and Direct Editing of Sickle Allele for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for the Treatment of Sickle Cell Disease

25. Preclinical and Clinical Demonstration of Immunogenicity by mRNA Vaccines against H10N8 and H7N9 Influenza Viruses

26. Characterization of humoral and cell-mediated immunity induced by mRNA vaccines expressing influenza hemagglutinin stem and nucleoprotein in mice and nonhuman primates

27. Uptake, Efficacy, and Systemic Distribution of Naked, Inhaled Short Interfering RNA (siRNA) and Locked Nucleic Acid (LNA) Antisense

28. Screening of Chemically Distinct Lipid Nanoparticles In Vivo Using DNA Barcoding Technology Towards Effectively Delivering Messenger RNA to Hematopoietic Stem and Progenitor Cells

29. Conversion of HbS to Hb G-Makassar By Adenine Base Editing Is Compatible with Normal Hemoglobin Function

30. 155 CD5 knockout enhances the potency of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for the treatment of T-cell malignancies

32. Evaluation of the Impact of a Naturally Occurring Beta Hemoglobin Variant, Hb G-Makassar, on Mature Red Blood Cell Function and Pathology in a Sickle Cell Disease Mouse Model

33. Non-Genotoxic Conditioning for Hematopoietic Stem Cell Transplant through Engineered Stem Cell Antibody Paired Evasion (ESCAPE)

34. Rationally Designed Base Editors for Precise Editing of the Sickle Cell Disease Mutation

35. Adenine Base Editing of the Sickle Allele in CD34+ Hematopoietic Stem and Progenitor Cells Eliminates Hemoglobin S

36. 111 Highly efficient multiplexed base editing enables development of universal CD7-targeting CAR-T Cells to treat T-ALL

37. Directed Evolution of Adenine Base Editors with Increased Activity and Therapeutic Application

41. Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

43. A Modified mRNA Vaccine Targeting Immunodominant NS Epitopes Protects Against Dengue Virus Infection in HLA Class I Transgenic Mice

44. A lipid-encapsulated mRNA encoding a potently neutralizing human monoclonal antibody protects against chikungunya infection

45. mRNA vaccines against H10N8 and H7N9 influenza viruses of pandemic potential are immunogenic and well tolerated in healthy adults in phase 1 randomized clinical trials

47. Corrigendum: Induction of Robust B Cell Responses After Influenza mRNA Vaccination Is Accompanied by Circulating Hemagglutinin-Specific ICOS+ PD-1+ CXCR3+ T Follicular Helper Cells

48. A multiclade env–gagVLP mRNA vaccine elicits tier-2 HIV-1-neutralizing antibodies and reduces the risk of heterologous SHIV infection in macaques

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