Human gene therapy

Keyphrases

Publications

volume 34, number 11-12, 2023

Alex Philippidis
Food and Drug Administration Sets Stage for Approval of First Duchenne Muscular Dystrophy Gene Therapy. Human gene therapy 34 (11-12) (2023)

volume 34, number 13-14, 2023

Hildegund Ertl
Rosalind Franklin Society Proudly Announces the 2022 Award Recipient for Human Gene Therapy . Human gene therapy 34 (13-14) (2023)
Alex Philippidis
Case Study Cites Immune Reaction to High Adeno-Associated Virus Dose in Explaining Duchenne Muscular Dystrophy Trial Death. Human gene therapy 34 (13-14) (2023)
Feng Gu
Advances in Split Vector Approaches for Adeno-Associated Virus Gene Therapy. Human gene therapy 34 (13-14) (2023)

volume 34, number 15-16, 2023

Alex Philippidis
BioMarin's ROCTAVIAN Wins Food and Drug Administration Approval As First Gene Therapy for Severe Hemophilia A. Human gene therapy 34 (15-16) (2023)

volume 34, number 17-18, 2023

Alex Philippidis
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family . Human gene therapy 34 (17-18) (2023)

volume 34, number 19-20, 2023

Alex Philippidis
Up to $1.5B in Milestones as Otsuka, Shape Partner on Eye Disease Gene Therapies. Human gene therapy 34 (19-20) (2023)

volume 34, number 21-22, 2023

Alex Philippidis
First Prime Editing Clinical Trial Expected in 2024. Human gene therapy 34 (21-22) (2023)
Terence R Flotte
Annual Editor's Letter. Human gene therapy 34 (21-22) (2023)

volume 34, number 3-4, 2023

Kenneth I Berns, Barry J Byrne, Terence R Flotte, R Jude Samulski, Arun Srivastava
Nicholas Muzyczka, PhD [1947-2023]. Human gene therapy 34 (3-4) (2023)
Zion Lee, Min Lu, Eesha Irfanullah, Morgan Soukup, Daniel Schmidt, Wei-Shou Hu
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors. Human gene therapy 34 (3-4) (2023)
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, Sébastien Monette, Ronald G Crystal, Dolan Sondhi
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 34 (3-4) (2023)
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
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 34 (3-4) (2023)
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
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 34 (3-4) (2023)
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, Sébastien Monette, Ronald G Crystal, Dolan Sondhi
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 34 (3-4) (2023)
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, Sébastien Monette, Ronald G Crystal, Dolan Sondhi
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 34 (3-4) (2023)
Zion Lee, Min Lu, Eesha Irfanullah, Morgan Soukup, Daniel Schmidt, Wei-Shou Hu
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors. Human gene therapy 34 (3-4) (2023)
Zion Lee, Min Lu, Eesha Irfanullah, Morgan Soukup, Daniel Schmidt, Wei-Shou Hu
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors. Human gene therapy 34 (3-4) (2023)
Kenneth I Berns, Barry J Byrne, Terence R Flotte, R Jude Samulski, Arun Srivastava
Nicholas Muzyczka, PhD [1947-2023]. Human gene therapy 34 (3-4) (2023)
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, Sébastien Monette, Ronald G Crystal, Dolan Sondhi
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 34 (3-4) (2023)
Lorraine Quillien, Louis Buscail, Pierre Cordelier
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future. Human gene therapy 34 (3-4) (2023)
Lorraine Quillien, Louis Buscail, Pierre Cordelier
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future. Human gene therapy 34 (3-4) (2023)
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, Sébastien Monette, Ronald G Crystal, Dolan Sondhi
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 34 (3-4) (2023)
Alex Philippidis
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease. Human gene therapy 34 (3-4) (2023)
Zion Lee, Min Lu, Eesha Irfanullah, Morgan Soukup, Daniel Schmidt, Wei-Shou Hu
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors. Human gene therapy 34 (3-4) (2023)
Xin Chen, Daniel A Lim, Michael W Lawlor, David Dimmock, Charles H Vite, Thomas Lester, Fatemeh Tavakkoli, Chanchal Sadhu, Suyash Prasad, Steven J Gray
Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration. Human gene therapy 34 (3-4) (2023)
Lorraine Quillien, Louis Buscail, Pierre Cordelier
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future. Human gene therapy 34 (3-4) (2023)
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, Sébastien Monette, Ronald G Crystal, Dolan Sondhi
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 34 (3-4) (2023)
Xin Chen, Daniel A Lim, Michael W Lawlor, David Dimmock, Charles H Vite, Thomas Lester, Fatemeh Tavakkoli, Chanchal Sadhu, Suyash Prasad, Steven J Gray
Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration. Human gene therapy 34 (3-4) (2023)
Alex Philippidis
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease. Human gene therapy 34 (3-4) (2023)
Alex Philippidis
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease. Human gene therapy 34 (3-4) (2023)
Xin Chen, Daniel A Lim, Michael W Lawlor, David Dimmock, Charles H Vite, Thomas Lester, Fatemeh Tavakkoli, Chanchal Sadhu, Suyash Prasad, Steven J Gray
Biodistribution of Adeno-Associated Virus Gene Therapy Following Cerebrospinal Fluid-Directed Administration. Human gene therapy 34 (3-4) (2023)
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
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 34 (3-4) (2023)

volume 34, number 5-6, 2023

Alex Philippidis
Magenta Halts Development, Pursues Strategic Alternatives After Patient Death. Human gene therapy 34 (5-6) (2023)
Yueyi Li, Hongyu Zeng, Yuhao Wei, Xuelei Ma, Zhiyao He
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy. Human gene therapy 34 (5-6) (2023)
Sebastian Schellhorn, Dominik Brücher, Natascha A Wolff, Katrin Schröer, Erwan Sallard, Kemal Mese, Wenli Zhang, Eric Ehrke-Schulz, Frank Thévenod, Andreas Plückthun, Anja Ehrhardt
Targeting Oncolytic Adenoviruses to Cancer Cells Using a Designed Ankyrin Repeat Protein Lipocalin-2 Fusion Protein. Human gene therapy 34 (5-6) (2023)
Yueyi Li, Hongyu Zeng, Yuhao Wei, Xuelei Ma, Zhiyao He
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy. Human gene therapy 34 (5-6) (2023)
Min Yu, Qian Zhang, Fancong Kong, Ling Qi, Yafang Pu, Lugui Qiu, Jian-Xiang Wang, Fei Li
Efficacy and Safety of Dual-Targeting Chimeric Antigen Receptor-T Therapy for Relapsed or Refractory B Cell Lymphoid Malignancies: A Systematic Review and Meta-Analysis. Human gene therapy 34 (5-6) (2023)
Tierney B Daw, Hala G El-Nahal, Michele A Basso, Elizabeth J Jun, Alex R Bautista, Richard Jude Samulski, Marc A Sommer, Martin O Bohlen
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain. Human gene therapy 34 (5-6) (2023)
Tierney B Daw, Hala G El-Nahal, Michele A Basso, Elizabeth J Jun, Alex R Bautista, Richard Jude Samulski, Marc A Sommer, Martin O Bohlen
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain. Human gene therapy 34 (5-6) (2023)
Richa Madan Lomash, Oleg Shchelochkov, Randy J Chandler, Charles P Venditti, Anne R Pariser, Elizabeth A Ottinger, null null
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience. Human gene therapy 34 (5-6) (2023)
Tierney B Daw, Hala G El-Nahal, Michele A Basso, Elizabeth J Jun, Alex R Bautista, Richard Jude Samulski, Marc A Sommer, Martin O Bohlen
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain. Human gene therapy 34 (5-6) (2023)
Houria Bachtarzi
Genome Editing: Moving Toward a New Era of Innovation, Development, and Approval. Human gene therapy 34 (5-6) (2023)
Richa Madan Lomash, Oleg Shchelochkov, Randy J Chandler, Charles P Venditti, Anne R Pariser, Elizabeth A Ottinger, null null
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience. Human gene therapy 34 (5-6) (2023)

volume 34, number 7-8, 2023

Alex Philippidis
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime. Human gene therapy 34 (7-8) (2023)
Alex Philippidis
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime. Human gene therapy 34 (7-8) (2023)
Alex Philippidis
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime. Human gene therapy 34 (7-8) (2023)
Alex Philippidis
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime. Human gene therapy 34 (7-8) (2023)

volume 34, number 9-10, 2023

Dongsheng Duan
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond. Human gene therapy 34 (9-10) (2023)
Alex Philippidis
CRISPR Therapeutics, Vertex Complete Rolling Biologics License Applications for Exa-Cel in Sickle Cell Disease, Beta Thalassemia. Human gene therapy 34 (9-10) (2023)