Molecular Therapy. Methods & Clinical Development
Overview
Molecular Therapy. Methods & Clinical Development is a scientific journal, published by Cell Press since 2014 in English. The journal's country of origin is United States.
Details
Details
Abbr.
Mol Ther Methods Clin Dev
Publisher
Cell Press
Start
2014
End
Continuing
e-ISSN
2329-0501
Country
United States
Language
English
Metrics
Metrics
h-index / Ranks: 6131
60
SJR / Ranks: 1302
1896
CiteScore / Ranks: 1650
9.40
JIF / Ranks: 1761
4.7
Recent Articles
1.
Xin D, Kurien L, Briggs K, Schimek A, Dambra R, Hochdorfer D, et al.
Mol Ther Methods Clin Dev
. 2025 Mar;
33(1):101429.
PMID: 40083959
Vesicular stomatitis virus expressing the glycoprotein of the lymphocytic choriomeningitis virus (VSV-GP) is a promising platform for oncolytic viruses and cancer vaccines. In this work, cryoelectron microscopy (cryo-EM) imaging was...
2.
Xu Y, Bai X, Lin J, Lu K, Weng S, Wu Y, et al.
Mol Ther Methods Clin Dev
. 2025 Mar;
33(1):101420.
PMID: 40034424
Recombinant adeno-associated virus (rAAV) is a widely used viral vector for gene therapy. However, a limitation of AAV-mediated gene therapy is that patients are typically dosed only once. In this...
3.
Diez B, Calvino C, Fernandez-Garcia M, Rodriguez-Marquez P, Rodriguez-Diaz S, Martinez-Turillas R, et al.
Mol Ther Methods Clin Dev
. 2025 Mar;
33(1):101425.
PMID: 40034423
Most CAR-T therapies rely on genetic T cell engineering with integrating viral vectors that, although effective, are associated with prohibitive costs. Here we have generated TranspoCART19 cells, a fully functional...
4.
Wang J, Cui M, Liu H, Guo P, McGowan J, Cheng S, et al.
Mol Ther Methods Clin Dev
. 2025 Mar;
33(1):101426.
PMID: 40027263
Recombinant adeno-associated virus (rAAV) is a leading vector for retinal gene therapy due to its favorable safety profile demonstrated by the FDA-approved Luxturna for Leber congenital amaurosis. However, challenges with...
5.
Svensson M, Limeres M, Zeyn Y, Gambaro R, Islan G, Berti I, et al.
Mol Ther Methods Clin Dev
. 2025 Mar;
33(1):101427.
PMID: 40027262
The liver, which plays pivotal roles in metabolism and immunity, often confers tolerance, suppressing immune responses to pathogens. Adjuvanted, lipid nanoparticle-encapsulated mRNA vaccines (mRNA-LNPs) offer a promising approach to overcome...
6.
Liu M, Cook E, Dai Y, Ehlert E, du Plessis F, Lubelski J, et al.
Mol Ther Methods Clin Dev
. 2025 Mar;
33(1):101411.
PMID: 40027261
Safely achieving therapeutic expression levels with adeno-associated virus (AAV) gene therapy is a significant challenge for treating the large muscle mass in humans. Non-human primates (NHPs) provide a more accurate...
7.
van Olden R, Lo Bianco C, Dilly K, Savelieva M, Xu S, Tijsma A, et al.
Mol Ther Methods Clin Dev
. 2025 Feb;
32(4):101344.
PMID: 40018025
Gene therapies such as onasemnogene abeparvovec for spinal muscular atrophy (SMA) utilize adeno-associated virus 9 (AAV9) for targeted gene delivery, which requires an AAV9 antibody (AAV9-Ab) immunoglobulin G (IgG) ≤1:50...
8.
Qiu B, Manzanares D, Li Y, Wang X, Li Z, Terreau S, et al.
Mol Ther Methods Clin Dev
. 2025 Feb;
32(3):101292.
PMID: 40017666
Lung cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF transmembrane conductance regulator () gene, leading to a dysfunctional CFTR protein. Gene therapy offers promise...
9.
Ables C, Jaramillo C, Wood E, Stern S, Alashari M, Book L, et al.
Mol Ther Methods Clin Dev
. 2025 Feb;
32(3):101296.
PMID: 40017665
Spinal muscular atrophy is a neurodegenerative disorder resulting from the irreversible loss of anterior horn cells secondary to homozygous mutations in the survival motor neuron gene . Gene replacement therapy...
10.
Pugliano C, Berger M, Ray R, Sapkos K, Wu B, Laird A, et al.
Mol Ther Methods Clin Dev
. 2025 Feb;
32(3):101297.
PMID: 40012884
Targeted gene editing to restore CD40L expression via homology-directed repair (HDR) in CD34 hematopoietic stem and progenitor cells (HSPCs) represents a potential long-term therapy for X-linked hyper IgM syndrome. However,...