Mol Ther Methods Clin Dev
Mol Ther Methods Clin Dev
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702
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Last Updated: Aug 05, 2025
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View All 702 PapersAAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia.
Chang JC, Ryan MR, Stark MC, Liu S, Purushothaman P, Bolan F, Johnson CA, Champe M, Meng H, Lawlor …
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and q…
Doshi BS, Samelson-Jones BJ, Nichols TC, Merricks EP, Siner JL, French RA, Lee BJ, Arruda VR, Calla…
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR.
Zhang J, Frabutt DA, Chrzanowski M, Li N, Miller LM, Tian J, Mulcrone PL, Lam AK, Draper BE, Jarrol…
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.
Rana J, Herzog RW, Muñoz-Melero M, Yamada K, Kumar SRP, Lam AK, Markusic DM, Duan D, Terhorst C, By…
Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor.
Stibbs DJ, Silva Couto P, Takeuchi Y, Rafiq QA, Jackson NB, Rayat ACME.
Extracellular vesicle depletion and UGCG overexpression mitigate the cell density effect in HEK293 cell culture transfe…
Pérez-Rubio P, Lavado-García J, Bosch-Molist L, Romero EL, Cervera L, Gòdia F.
Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice.
Lin Y, Kang Z, Su C, Li S, Xie W.
High-titer manufacturing of SARS-CoV-2 Spike-pseudotyped VSV in stirred-tank bioreactors.
Todesco HM, Gafuik C, John CM, Roberts EL, Borys BS, Pawluk A, Kallos MS, Potts KG, Mahoney DJ.
Liter-scale manufacturing of shelf-stable plasmid DNA/PEI transfection particles for viral vector production.
Hu Y, Eder BA, Lin J, Li S, Zhu Y, Wang TH, Guo T, Mao HQ.
Long-term stability of clinical-grade lentiviral vectors for cell therapy.
Jadlowsky JK, Leskowitz R, McKenna S, Karar J, Ma Y, Dai A, Plesa G, Chen F, Alexander K, Petrella …