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Molecular therapy : the journal of the American Society of Gene Therapy.

Periodical
Molecular Biology
Therapeutics
Gene Transfer Techniques
Genetic Therapy
Publisher:
Academic Press,. Cambridge, MA : Cell Press (2017)
Frequency: Monthly
Country: United States
Language: English
Author(s):
American Society of Gene Therapy.
Start Year:2000 -
ISSN:
1525-0016 (Print)
1525-0024 (Electronic)
1525-0016 (Linking)
Impact Factor
12.4
2022
NLM ID:100890581
(OCoLC):41354718
LCCN:sn 99008627
Classification:W1 MO1975M
Nebulization of an mRNA-encoded monoclonal antibody for passive immunization of foals against Rhodococcus equi.
Molecular therapy : the journal of the American Society of Gene Therapy    June 14, 2025   doi: 10.1016/j.ymthe.2025.06.025
Legere RM, Ott JA, Poveda C, Vanover D, Borba KER, Yeon Joo J, Martin CL, da Silveira BP, Bray JM, Landrock K, Wright GA, Blazier JC, Hillhouse AE....Inhalation of Rhodococcus equi causes severe pneumonia in humans and animals worldwide, most commonly affecting horse foals. The standard for preventing R. equi pneumonia in foals is transfusion of hyperimmune plasma, which is expensive and carries the risk of adverse effects. Our goal was to passively immunize foals against R. equi by nebulizing mRNA encoding an equine monoclonal antibody (mAb) against the virulence-associated protein A (VapA) directly into the lungs. VapA-specific memory B cells from an immunized horse were used to identify and select the sequence for an equine immunoglobuli...
MicroRNA29a Treatment Improves Early Tendon Injury.
Molecular therapy : the journal of the American Society of Gene Therapy    July 28, 2017   Volume 25, Issue 10 2415-2426 doi: 10.1016/j.ymthe.2017.07.015
Watts AE, Millar NL, Platt J, Kitson SM, Akbar M, Rech R, Griffin J, Pool R, Hughes T, McInnes IB, Gilchrist DS.Tendon injuries (tendinopathies) are common in human and equine athletes and characterized by dysregulated collagen matrix, resulting in tendon damage. We have previously demonstrated a functional role for microRNA29a (miR29a) as a post-transcriptional regulator of collagen 3 expression in murine and human tendon injury. Given the translational potential, we designed a randomized, blinded trial to evaluate the potential of a miR29a replacement therapy as a therapeutic option to treat tendinopathy in an equine model that closely mimics human disease. Tendon injury was induced in the superficial...
Implantation of rAAV5-IGF-I transduced autologous chondrocytes improves cartilage repair in full-thickness defects in the equine model.
Molecular therapy : the journal of the American Society of Gene Therapy    October 14, 2014   Volume 23, Issue 2 363-373 doi: 10.1038/mt.2014.198
Ortved KF, Begum L, Mohammed HO, Nixon AJ.Cartilage injury often precipitates osteoarthritis which has driven research to bolster repair in cartilage impact damage. Autologous chondrocytes transduced with rAAV5-IGF-I were evaluated in chondral defects in a well-established large animal model. Cartilage was harvested from the talus of 24 horses; chondrocytes were isolated and stored frozen. Twenty million cells were cultured and transduced with 10(5) AAV vg/cell prior to implantation. Chondrocytes from eight horses were transduced with rAAV5-IGF-I, chondrocytes from eight horses with rAAV5-GFP, and chondrocytes from eight horses were n...