Human Gene Therapy
Overview
Human Gene Therapy is a renowned scientific journal that focuses on cutting-edge research and advancements in the field of gene therapy. It covers a wide range of topics, including gene delivery systems, clinical trials, gene editing technologies, and therapeutic applications. With its rigorous peer-review process, the journal serves as a valuable resource for scientists, clinicians, and researchers interested in the latest developments and potential of gene therapy for treating various genetic disorders and diseases.
Details
Details
Abbr.
Hum Gene Ther
Start
1990
End
Continuing
Frequency
Twelve no. a year, 2004-
p-ISSN
1043-0342
e-ISSN
1557-7422
Country
United States
Language
English
Specialties
Pharmacology
Genetics
Genetics
Metrics
Metrics
h-index / Ranks: 1255
159
SJR / Ranks: 3270
1091
CiteScore / Ranks: 2894
7.00
JIF / Ranks: 2095
4.2
Recent Articles
21.
Ozgur Gunes Y, Le Stunff C, Bougneres P
Hum Gene Ther
. 2024 Dec;
36(3-4):88-100.
PMID: 39723977
Worldwide, thousands of male patients who carry ATP Binding Cassette Subfamily D Member 1 () mutations develop adrenomyeloneuropathy (AMN) in mid-adulthood, a debilitating axonopathy of the spinal cord. Today AAV...
22.
Lehman S, Vreeland W, Blaszczyk A, Adams-Hall S, Ahuja S, Arnaout A, et al.
Hum Gene Ther
. 2024 Dec;
36(1-2):36-44.
PMID: 39723438
Recombinant adeno-associated virus (AAV) is one of the main viral vector-based gene therapy platforms. AAV is a virus consisting of a ≈25 nm diameter capsid with a ≈4.7 kb cargo...
23.
Piccolo P, Brunetti-Pierri N
Hum Gene Ther
. 2024 Dec;
36(3-4):77-87.
PMID: 39714937
Adeno-associated virus (AAV) vectors have demonstrated safety and efficacy for gene transfer to hepatocytes in preclinical models, in various clinical trials and from a clinical experience with a growing number...
24.
25.
Nagy A, Eichler F, Bley A, Bredow J, Fay A, Townsend E, et al.
Hum Gene Ther
. 2024 Dec;
36(1-2):45-56.
PMID: 39628365
Canavan disease (CD) is an ultra-rare autosomal recessive leukodystrophy caused by loss-of-function mutations in which encodes aspartoacylase (ASPA), leading to accumulation of -acetylaspartate (NAA). Patients with CD typically present with...
26.
Baine S, Wier C, Lemmerman L, Cooper-Olson G, Kempton A, Haile A, et al.
Hum Gene Ther
. 2024 Nov;
35(23-24):978-988.
PMID: 39607794
Delandistrogene moxeparvovec is a gene transfer therapy for Duchenne muscular dystrophy (DMD) that uses an adeno-associated viral vector to deliver a micro-dystrophin transgene to skeletal and cardiac muscle. This study...
27.
Qin X, Li H, Zhao H, Xiang K, Liu S, Lou R, et al.
Hum Gene Ther
. 2024 Nov;
35(23-24):969-977.
PMID: 39607725
To facilitate adeno-associated virus (AAV)-mediated gene therapy in China, we conducted a study on the distribution of AAV-neutralizing antibodies (NAbs) in healthy subjects and in patients with Duchenne muscular dystrophy...
28.
Zappala A, Li H, Inoue K
Hum Gene Ther
. 2024 Nov;
35(23-24):989-999.
PMID: 39585211
Systemic delivery of adeno-associated virus (AAV) vectors targeting the central nervous system has the potential to solve many neurodevelopmental disorders, yet it is made difficult by the filtering effect of...
29.
Flynn K, Jussila D
Hum Gene Ther
. 2024 Nov;
35(21-22):869-881.
PMID: 39570090
No abstract available.
30.