Human Gene Therapy
@HGTJournal
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The first journal devoted to the science, technology and clinical application of human gene therapy. Established in 1990. Editor-in-Chief: Terry Flotte.
New Rochelle, New York
Joined February 2016
Read the most downloaded articles of 2024, free through February 27th, including: Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article (Open Access)
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Read the most downloaded articles of 2024, free through February 27th, including: Prevalence of Adeno-Associated Virus-9-Neutralizing Antibody in Chinese Patients with #DuchenneMuscularDystrophy
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Read the most downloaded articles of 2024, free through February 27th, including: Complement System Response to Adeno-Associated Virus Vector #GeneTherapy:
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New Issue Alert -- Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases; Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated #GeneTherapy to the Central Nervous System for CLN2 Disease; and more!
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Human Gene Therapy Editor-in-Chief Terence Flotte, MD, is a featured speaker at @GENbio's Second Annual Summit highlighting The State of Cell and Gene Therapy. Register free now to hear him and all of the summit's guest speakers:
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A new ferret model of cystic fibrosis lights up CFTR-expressing cells and allows for targeted genetic rescue. Learn more:
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Hear from Michel Sadelain in his "CARs in 2025: the Cambrian Explosion Continues" keynote at the State of Cell and Gene Therapy summit happening January 29th To register for FREE, visit:
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Ambros and Ruvkun’s Nobel Prize-winning #miRNA discovery has provided another tool in the gene therapy arsenal. Read the article today:
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Read now -- Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy:
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Read now: Gene Therapy to Treat Chronic Hypereosinophilia.
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Free through January 6: Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice.
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Free Access -- Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMDMDX Rats:
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Long-Term Survival and Cardiac Efficacy of a #GeneTherapy for #Duchenne Muscular Dystrophy -- Ahead of Print in @HGTjournal:
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Newly published in @HGTJournal -- Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMDMDX Rats:
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Unconstrained mitochondrial base editing with αDdCBEs: Challenging and circumventing the TALE 5'-T constraint. Read the full Open Access article today:
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Discover key insights into leading microRNA-based therapeutics in preclinical and clinical studies. Read the article:
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Recent advancements in lipid nanoparticles engineering for extra-hepatic delivery of nucleic acids therapeutics. Read the article:
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Spirovant Sciences is proud to share the publication of 2 manuscripts in Human Gene Therapy that outline non-clinical work supporting the initiation of our clinical trial for cystic fibrosis. Thanks to all who contributed to this work. #CureCF Read more:
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Ha et al. show the potential of an IL7RA-SCID gene therapy using a SIN-alpharetroviral vector in a mouse model. Read more:
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Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair:
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