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
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Mylvara A, Davidson C, Alexander D, Venditti C, Porter F
Hum Gene Ther
. 2025 Feb;
PMID: 40014475
Niemann-Pick disease, type C1 (NPC1), is a rare, fatal neurodegenerative disorder caused by pathological variations in . We and others have previously demonstrated the efficacy of systemic adeno-associated virus (AAV)...
3.
Chen G, Zhan X, Gao X, Yi M, Liang H, Chen Y, et al.
Hum Gene Ther
. 2025 Feb;
PMID: 40014367
Mucopolysaccharidosis type II (MPS II) is an X-linked lysosomal storage disorder caused by pathogenic variants in the gene encoding iduronate-2-sulfatase (IDS), which hydrolyzes sulfate groups in dermatan sulfate and heparan...
4.
Hinsch V, Boye S, Boye S
Hum Gene Ther
. 2025 Feb;
PMID: 39989340
The eye is an ideal target for gene therapy due its accessibility, immune privilege, small size, and compartmentalization. Adeno-associated virus (AAV) is the gold standard vector for gene delivery and...
5.
Carisi M, Shamber C, Bishop M, Sangster M, Chandrachud U, Meyerink B, et al.
Hum Gene Ther
. 2025 Feb;
36(5-6):637-652.
PMID: 39978419
Beta-propeller protein-associated neurodegeneration (BPAN) is an ultra-rare, X-linked dominant, neurodevelopmental, and neurodegenerative disease caused by loss-of-function mutations in the gene. It manifests in neurodevelopmental delay and seizures followed by secondary...
6.
Lomash R, Dehdashti J, Shchelochkov O, Chandler R, Li L, Manoli I, et al.
Hum Gene Ther
. 2025 Feb;
36(5-6):653-662.
PMID: 39976996
Gene therapy development presents multiple challenges, and early planning is vital in the successful implementation of such programs. The Platform Vector Gene Therapy (PaVe-GT) program is a National Institutes of...
7.
Suleman S, Khalifa M, Fawaz S, Alhaque S, Chinea Y, Themis M
Hum Gene Ther
. 2025 Feb;
36(5-6):628-636.
PMID: 39973307
Lentivirus (LV) vectors offer permanent delivery of therapeutic genes to the host through an RNA intermediate genome. They are one of the most commonly used vectors for clinical gene therapy...
8.
Gibson W, Lengyell T, Korecki A, Janssen S, Adair B, Gamu D, et al.
Hum Gene Ther
. 2025 Feb;
36(5-6):618-627.
PMID: 39964768
Weaver syndrome is a rare neurodevelopmental disorder that encompasses macrocephaly, tall stature, obesity, brain anomalies, intellectual disability, and increased susceptibility to cancer. This dominant monogenic disorder is caused by germline...
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