Aspects of Gene Therapy Products Using Current Genome-Editing Technology in Japan
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 17, 2020 Category: Genetics & Stem Cells Authors: Teruhide Yamaguchi Eriko Uchida Takashi Okada Keiya Ozawa Masafumi Onodera Akihiro Kume Takashi Shimada Satoru Takahashi Kenzaburo Tani Yasutomo Nasu Tomoji Mashimo Hiroyuki Mizuguchi Kohnosuke Mitani Kazushige Maki Source Type: research

Transduction of Surface and Basal Cells in Rhesus Macaque Lung Following Repeat Dosing with AAV1CFTR
Human Gene Therapy,Volume 31, Issue 17-18, Page 1010-1023, September 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: William B. Guggino Murali K. Yanda Cristina V. Cebotaru Liudmila Cebotaru Source Type: research

New Directions in Pulmonary Gene Therapy
Human Gene Therapy,Volume 31, Issue 17-18, Page 921-939, September 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: Amber Vu Paul B. McCray Source Type: research

Gene Therapy for Respiratory Diseases: Progress and a Changing Context
Human Gene Therapy,Volume 31, Issue 17-18, Page 911-916, September 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: Eric W.F.W. Alton A. Christopher Boyd Jane C. Davies Deborah R. Gill Uta Griesenbach Tracy E. Harman Stephen Hyde Gerry McLachlan Source Type: research

After Third Death, Audentes' AT132 Remains on Clinical Hold
Human Gene Therapy,Volume 31, Issue 17-18, Page 908-910, September 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: Alex Philippidis Source Type: research

Call for Special Issue Papers: Oncolytic Viruses and Cancer Immunotherapy
Human Gene Therapy,Volume 31, Issue 17-18, Page 907-907, September 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: Prem Seth Source Type: research

Breathing Easier: Molecular Therapeutics for Cystic Fibrosis
Human Gene Therapy,Volume 31, Issue 17-18, Page 905-906, September 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 16, 2020 Category: Genetics & Stem Cells Authors: Maria P. Limberis Source Type: research

Laying the Foundation for Cystic Fibrosis Therapy: An Interview with Michael Boyle
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 11, 2020 Category: Genetics & Stem Cells Authors: Kevin Davies Source Type: research

Novel Lung Tropic Adeno-Associated Virus Capsids for Therapeutic Gene Delivery
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 8, 2020 Category: Genetics & Stem Cells Authors: Ana Carneiro Hyuncheol Lee Li Lin Joost van Haasteren David V. Schaffer Source Type: research

Correction of Airway Stem Cells: Genome Editing Approaches for the Treatment of Cystic Fibrosis
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 8, 2020 Category: Genetics & Stem Cells Authors: Nicholas E. King Shingo Suzuki Cristina Barill à Finn J. Hawkins Scott H. Randell Susan D. Reynolds Barry R. Stripp Brian R. Davis Source Type: research

Will Airway Gene Therapy for Cystic Fibrosis Improve Lung Function? New Imaging Technologies Can Help Us Find Out
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 8, 2020 Category: Genetics & Stem Cells Authors: David Parsons Martin Donnelley Source Type: research

Treating Cystic Fibrosis with mRNA and CRISPR
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - September 8, 2020 Category: Genetics & Stem Cells Authors: Alejandro Da Silva Sanchez Kalina Paunovska Ana Cristian James E. Dahlman Source Type: research

Adeno-Associated Virus-Based Gene Therapy for Lifelong Correction of Genetic Disease
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 21, 2020 Category: Genetics & Stem Cells Authors: Christian M. Brommel Ashley L. Cooney Patrick L. Sinn Source Type: research

Gene Editing for Treatment and Prevention of Human Diseases: A Global Survey of Gene Editing-Related Researchers
Human Gene Therapy,Volume 31, Issue 15-16, Page 852-862, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Leonardo Fernandes Moutinho Rocha Luiza Amara Maciel Braga Fabio Batista Mota Source Type: research

Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy
Human Gene Therapy,Volume 31, Issue 15-16, Page 819-827, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Christiana O. Salami Katie Jackson Clarisse Jose Laith Alyass Georges-Ibrahim Cisse Bishnu P. De Katie M. Stiles Maria J. Chiuchiolo Dolan Sondhi Ronald G. Crystal Stephen M. Kaminsky Source Type: research

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
Human Gene Therapy,Volume 31, Issue 15-16, Page 808-818, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Juliette Hordeaux Elizabeth L. Buza Cecilia Dyer Tamara Goode Thomas W. Mitchell Laura Richman Nathan Denton Christian Hinderer Nathan Katz Ralf Schmid Rod Miller Gourav R. Choudhury Makoto Horiuchi Kalyani Nambiar Hanying Yan Mingyao Li James M. Wilson Source Type: research

BioMarin Defends Up-to-$3M List Price of Hemophilia A Gene Therapy
Human Gene Therapy,Volume 31, Issue 15-16, Page 791-793, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Alex Philippidis Source Type: research

Call for Special Issue Papers: Oncolytic Viruses and Cancer Immunotherapy
Human Gene Therapy,Volume 31, Issue 15-16, Page 790-790, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Source Type: research

Call for Papers: Applications of Next Generation CRISPR-Derived Technologies
Human Gene Therapy,Volume 31, Issue 15-16, Page 789-789, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Source Type: research

Call for Submissions for The 2020 European Society of Gene and Cell Therapy (ESGCT) Special Issue: Gene and Cell Therapy for Inherited and Acquired Immune Deficiency
Human Gene Therapy,Volume 31, Issue 15-16, Page 788-788, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Source Type: research

Re: “Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy” by Wilson and Flotte
Human Gene Therapy,Volume 31, Issue 15-16, Page 787-787, August 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Perry B. Shieh Carsten G. B önnemann Wolfgang M üller-Felber Astrid Blaschek James J. Dowling Nancy L. Kuntz Andreea M. Seferian Source Type: research

Design and Rationale for First-in-Human Phase 1 Immunovirotherapy Clinical Trial of Oncolytic HSV G207 to Treat Malignant Pediatric Cerebellar Brain Tumors
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Joshua D. Bernstock Asim K. Bag John Fiveash Kara Kachurak Galal Elsayed Gustavo Chagoya Florian Gessler Pablo A. Valdes Avi Madan-Swain Richard Whitley James M. Markert G. Yancey Gillespie James M. Johnston Gregory K. Friedman Source Type: research

Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Harrison C. Brown Christopher B. Doering Roland W. Herzog Chen Ling David M. Markusic H. Trent Spencer Alok Srivastava Arun Srivastava Source Type: research

Chorioallantoic Membrane Tumor Model for Evaluating Oncolytic Viruses
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - August 17, 2020 Category: Genetics & Stem Cells Authors: Lea Krutzke Ellen Allmendinger Katja Hirt Stefan Kochanek Source Type: research

Laying the Foundation for Neuromuscular Disease Gene Therapy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 28, 2020 Category: Genetics & Stem Cells Authors: Dongsheng Duan Source Type: research

CRISPR/Cas9 Editing: Sparking Discussion on Safety in Light of the Need for New Therapeutics
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 24, 2020 Category: Genetics & Stem Cells Authors: Tiffany M. Carlaw Lin-Hua Zhang Colin J.D. Ross Source Type: research

Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 17, 2020 Category: Genetics & Stem Cells Authors: Karina Krotova George Aslanidi Source Type: research

Engineered Capsids for Efficient Gene Delivery to the Retina and Cornea
Human Gene Therapy,Volume 31, Issue 13-14, Page 756-774, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Authors: Amy Frederick Jennifer Sullivan Lin Liu Matthew Adamowicz Michael Lukason Jasmine Raymer Zhengyu Luo Xiaoying Jin Kollu Nageswara Rao Catherine O'Riordan Source Type: research

Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease
Human Gene Therapy,Volume 31, Issue 13-14, Page 709-718, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Authors: Sophia-Martha kleine Holthaus Mikel Aristorena Ryea Maswood Olha Semenyuk Justin Hoke Aura Hare Alexander J. Smith Sara E. Mole Robin R. Ali Source Type: research

Pfizer and Sangamo Hemophilia A Gene Therapy Aces First Clinical Test
Human Gene Therapy,Volume 31, Issue 13-14, Page 705-708, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Authors: Alex Philippidis Source Type: research

Call for Papers: Applications of Next Generation CRISPR-Derived Technologies
Human Gene Therapy,Volume 31, Issue 13-14, Page 703-703, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Source Type: research

Retinal Gene Therapy: Expansion in Clinical Trials Drives the Need for Further Preclinical Research
Human Gene Therapy,Volume 31, Issue 13-14, Page 701-702, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Authors: Robin R. Ali Source Type: research

Black Lives Matter to Gene Therapy
Human Gene Therapy,Volume 31, Issue 13-14, Page 700-700, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Authors: Terence R. Flotte Jennifer E. Adair Source Type: research

AAV Vectors: Are They Safe?
Human Gene Therapy,Volume 31, Issue 13-14, Page 697-699, July 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 15, 2020 Category: Genetics & Stem Cells Authors: Arun Srivastava Source Type: research

Quantification of Adeno-Associated Virus with Safe Nucleic Acid Dyes
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 14, 2020 Category: Genetics & Stem Cells Authors: Jian Xu Steven H. DeVries Yongling Zhu Source Type: research

Intralingual Administration of AAVrh10-miRSOD1 Improves Respiratory But Not Swallowing Function in a Superoxide Dismutase-1 Mouse Model of Amyotrophic Lateral Sclerosis
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 13, 2020 Category: Genetics & Stem Cells Authors: Lori A. Lind Ellyn M. Andel Angela L. McCall Justin S. Dhindsa Katherine A. Johnson Olivia E. Stricklin Christian Mueller Mai K. ElMallah Teresa E. Lever Nicole L. Nichols Source Type: research

Defining Transcription Regulatory Elements in the Human Frataxin Gene: Implications for Gene Therapy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 13, 2020 Category: Genetics & Stem Cells Authors: Jixue Li Yanjie Li Jun Wang Trevor J. Gonzalez Aravind Asokan Jill S. Napierala Marek Napierala Source Type: research

Evaluation of Photoreceptor Transduction Efficacy of Capsid-Modified Adeno-Associated Viral Vectors Following Intravitreal and Subretinal Delivery in Sheep
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 6, 2020 Category: Genetics & Stem Cells Authors: Maya Ross Alexey Obolensky Edward Averbukh Raaya Ezra-Elia Esther Yamin Hen Honig Hay Dvir Alexander Rosov William W. Hauswirth Elisha Gootwine Eyal Banin Ron Ofri Source Type: research

Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - July 1, 2020 Category: Genetics & Stem Cells Authors: James M. Wilson Terence R. Flotte Source Type: research

Oncolytic Adenovirus Expressing ST13 Increases Antitumor Effect of Tumor Necrosis Factor-Related Apoptosis-Inducing Ligand Against Pancreatic Ductal Adenocarcinoma
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 30, 2020 Category: Genetics & Stem Cells Authors: Youni Zhang Miaojuan Ye Fang Huang Shibing Wang Huiju Wang Xiaozhou Mou Yigang Wang Source Type: research

Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 29, 2020 Category: Genetics & Stem Cells Authors: Chunjuan Song Val érie L. Dufour Artur V. Cideciyan Guo-Jie Ye Malgorzata Swider Judith A. Newmark Adrian M. Timmers Paulette M. Robinson David R. Knop Jeffrey D. Chulay Samuel G. Jacobson Gustavo D. Aguirre William A. Beltran Mark S. Shearman Source Type: research

Effect of AAV-Mediated Rhodopsin Gene Augmentation on Retinal Degeneration Caused by the Dominant P23H Rhodopsin Mutation in a Knock-In Murine Model
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 26, 2020 Category: Genetics & Stem Cells Authors: Harry O. Orlans Alun R. Barnard Maria I. Patr ício Michelle E. McClements Robert E. MacLaren Source Type: research

Correction to: Toxicity and Efficacy Evaluation of an Adeno-Associated Virus Vector Expressing Codon-Optimized RPGR Delivered by Subretinal Injection in a Canine Model of X-linked Retinitis Pigmentosa by Dufour VL, et al. Hum Gene Ther 2020;31:253 –267. DOI: 10.1089/hum.2019.297
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 25, 2020 Category: Genetics & Stem Cells Source Type: research

Call for Special Issue Papers: Oncolytic Viruses and Cancer Immunotherapy
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 19, 2020 Category: Genetics & Stem Cells Authors: Prem Seth Source Type: research

Gene Therapy for Hepatocellular Carcinoma Using Adenoviral Vectors Delivering a Gene Encoding IL-17A-Neutralizing Antibody Fragments
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 16, 2020 Category: Genetics & Stem Cells Authors: Haoyu Zou Israth Jahan Tuhin Masuma Akter Monty Shenggen Luo Jiaqi Shao Zhiqiang Yan Lei Yu Source Type: research

Lentiviral Gene Therapy for Familial Hemophagocytic Lymphohistiocytosis Type 3, Caused by UNC13D Genetic Defects
Human Gene Therapy,Volume 31, Issue 11-12, Page 626-638, June 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 12, 2020 Category: Genetics & Stem Cells Authors: Sarah E. Takushi Na Yoon Paik Andrew Fedanov Chengyu Prince Christopher B. Doering H. Trent Spencer Shanmuganathan Chandrakasan Source Type: research

Call for Papers: Applications of Next Generation CRISPR-Derived Technologies
Human Gene Therapy,Volume 31, Issue 11-12, Page 610-610, June 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 12, 2020 Category: Genetics & Stem Cells Source Type: research

LyP-1-Modified Oncolytic Adenoviruses Targeting Transforming Growth Factor β Inhibit Tumor Growth and Metastases and Augment Immune Checkpoint Inhibitor Therapy in Breast Cancer Mouse Models
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 12, 2020 Category: Genetics & Stem Cells Authors: Weidong Xu Yuefeng Yang Zebin Hu Maria Head Kathy A. Mangold Megan Sullivan Edward Wang Poornima Saha Kamalakar Gulukota Donald L. Helseth Theresa Guise Bellur S. Prabhkar Karen Kaul Hans Schreiber Prem Seth Source Type: research

Single-Cell Proteomics Reveals Immune Persistence in Cutting-Edge CAR-T Therapies
Human Gene Therapy,Volume 31, Issue 11-12, Page 692-693, June 2020. (Source: Human Gene Therapy)
Source: Human Gene Therapy - June 11, 2020 Category: Genetics & Stem Cells Source Type: research

Volume and Infusion Rate Dynamics of Intraparenchymal Central Nervous System Infusion in a Large Animal Model
Human Gene Therapy, Ahead of Print. (Source: Human Gene Therapy)
Source: Human Gene Therapy - May 29, 2020 Category: Genetics & Stem Cells Authors: Toloo Taghian Erin Horn Mohammed Salman Shazeeb Lindsey J. Bierfeldt Susan M. Tuominen Jennifer Koehler Deborah Fernau Stephanie Bertrand Stephen Frey Oguz I. Cataltepe Matthew J. Gounis Aly H. Abayazeed Terence R. Flotte Miguel Sena-Esteves Heather L. Gr Source Type: research