Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - March 14, 2023 Category: Genetics & Stem Cells Authors: Jeffrey S. Chamberlain Melissa Robb Serge Braun Kristy J. Brown Olivier Danos Annie Ganot Pedro Gonzalez-Alegre Nina Hunter Craig McDonald Carl Morris Mark Tobolowsky Kathryn R. Wagner Olivia Ziolkowski Dongsheng Duan Source Type: research
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - March 10, 2023 Category: Genetics & Stem Cells Authors: Da-Lu Wang Qi Su Qiao Qu Zhi-Long Li Zhen Liu Source Type: research
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - March 7, 2023 Category: Genetics & Stem Cells Authors: Tierney B. Daw Hala G. El-Nahal Michele A. Basso Elizabeth J. Jun Alex R. Bautista R. Jude Samulski Marc A. Sommer Martin O. Bohlen Source Type: research
T Cell Responses to Dystrophin in a Natural History Study of Duchenne Muscular Dystrophy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 24, 2023 Category: Genetics & Stem Cells Authors: Karen Anthony Pierpaolo Ala Francesco Catapano Jinhong Meng Joana Domingos Mark Perry Valeria Ricotti Kate Maresh Lauren C. Phillips Laurent Servais Andreea M. Seferian Silvana De Lucia Imelda de Groot Yvonne D. Krom J.G.M. Verschuuren Erik H. Niks Volker Source Type: research
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors
Human Gene Therapy,Volume 34, Issue 3-4, Page 162-170, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Zion Lee Min Lu Eesha Irfanullah Morgan Soukup Daniel Schmidt Wei-Shou Hu Source Type: research
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future
Human Gene Therapy,Volume 34, Issue 3-4, Page 150-161, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Lorraine Quillien Louis Buscail Pierre Cordelier Source Type: research
Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency
Human Gene Therapy,Volume 34, Issue 3-4, Page 139-149, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Jonathan B. Rosenberg Bishnu P. De Alessandria Greco Nicholas Gorman Vikrum Kooner Alvin Chen Melissa Yost-Bido Carlos Munoz-Zuluaga Stephen M. Kaminsky Mahboubeh Rostami S ébastien Monette Ronald G. Crystal Dolan Sondhi Source Type: research
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis
Human Gene Therapy,Volume 34, Issue 3-4, Page 129-138, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Carlos Pascual-Morena Iv án Cavero-Redondo Maribel Lucer ón-Lucas-Torres Irene Mart ínez-García Eva Rodr íguez-Gutiérrez Vicente Mart ínez-Vizcaíno Source Type: research
Advances in Off-Target Detection for CRISPR-Based Genome Editing
Human Gene Therapy,Volume 34, Issue 3-4, Page 112-128, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Haozheng Wang Yangmin Wang Zhongtao Luo Xinjian Lin Meilin Liu Fenglin Wu Hongwei Shao Wenfeng Zhang Source Type: research
Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration
Human Gene Therapy,Volume 34, Issue 3-4, Page 94-111, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Xin Chen Daniel A. Lim Michael W. Lawlor David Dimmock Charles H. Vite Thomas Lester Fatemeh Tavakkoli Chanchal Sadhu Suyash Prasad Steven J. Gray Source Type: research
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease
Human Gene Therapy,Volume 34, Issue 3-4, Page 90-93, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Alex Philippidis Source Type: research
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop
Human Gene Therapy,Volume 34, Issue 3-4, Page 83-89, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 14, 2023 Category: Genetics & Stem Cells Authors: Cheryl L. McDonald Pankaj Qasba Daniel G. Anderson Gang Bao Richard A. Colvin Donald B. Kohn Punam Malik Michael J. Mitchell William T. Pu David J. Rawlings David A. Williams Terence R. Flotte Source Type: research
Nicholas Muzyczka, PhD [1947 –2023]
Human Gene Therapy,Volume 34, Issue 3-4, Page 81-82, February 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - February 13, 2023 Category: Genetics & Stem Cells Authors: Kenneth I. Berns Barry J. Byrne Terence R. Flotte R. Jude Samulski Arun Srivastava Source Type: research
Quantitation of Coxsackievirus A21 Viral Proteins in Mixtures of Empty and Full Capsids Using Capillary Western
Human Gene Therapy,Volume 34, Issue 1-2, Page 68-77, January 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 17, 2023 Category: Genetics & Stem Cells Authors: Paul F. Gillespie Richard R. Rustandi Andrew R. Swartz Liang Shang Jessica Raffaele Ashley Prout Nicholas Cunningham Mohamed Dawod James Z. Deng Shiyi Wang Jessica Olson Yvonne Shieh John W. Loughney Source Type: research
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity
Human Gene Therapy,Volume 34, Issue 1-2, Page 56-67, January 2023. (Source: Human Gene Therapy)
Source: Human Gene Therapy - January 17, 2023 Category: Genetics & Stem Cells Authors: Huiren Zhao W. Hans Meisen Songli Wang Ki Jeong Lee Source Type: research
