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Gene therapy

Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia

Xufeng Dai, Ying He, [ ... ], Ji-jing Pang

Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye

Li Wang, Ru Xiao, Eva Andres-Mateos, Luk H. Vandenberghe

Genetically engineered suicide gene in mesenchymal stem cells using a Tet-On system for anaplastic thyroid cancer

Senthilkumar Kalimuthu, Ji Min Oh, [ ... ], Byeong-Cheol Ahn

Therapeutic efficacy of AAV8-mediated intrastriatal delivery of human cerebral dopamine neurotrophic factor in 6-OHDA-induced parkinsonian rat models with different disease progression

Lizheng Wang, Zixuan Wang, [ ... ], Xianghui Yu

Biodegradable poly (lactic acid-co-glycolic acid) scaffolds as carriers for genetically-modified fibroblasts

Tatjana Perisic, Ziyang Zhang, [ ... ], Arndt F. Schilling

Modeling timelines for translational science in cancer; the impact of technological maturation

Laura M. McNamee, Fred D. Ledley

Targeted AAV5-Smad7 gene therapy inhibits corneal scarring in vivo

Suneel Gupta, Jason T. Rodier, [ ... ], Rajiv R. Mohan

Targeting gene expression to specific cells of kidney tubules in vivo, using adenoviral promoter fragments

Sumiyo Watanabe, Toru Ogasawara, [ ... ], Shunya Uchida

Self-complementary adeno-associated virus serotype 6 mediated knockdown of ADAMTS4 induces long-term and effective enhancement of aggrecan in degenerative human nucleus pulposus cells: A new therapeutic approach for intervertebral disc disorders

Demissew Shenegelegn Mern, Anja Tschugg, Sebastian Hartmann, Claudius Thomé

Simultaneous Administration of ADSCs-Based Therapy and Gene Therapy Using Ad-huPA Reduces Experimental Liver Fibrosis

Alejandra Meza-Ríos, Leonel García-Benavides, [ ... ], Ana Sandoval-Rodríguez

Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma

Martin J. Hicks, Maria J. Chiuchiolo, [ ... ], Ronald G. Crystal

Insulin-Like Growth Factor I (IGF-I) Expressed from an AAV1 Vector Leads to a Complete Reversion of Liver Cirrhosis in Rats

Luciano Sobrevals, Mónica Enguita, [ ... ], Puri Fortes

Gene Therapy Corrects Mitochondrial Dysfunction in Hematopoietic Progenitor Cells and Fibroblasts from Coq9R239X Mice

Eliana Barriocanal-Casado, Cristina Cueto-Ureña, [ ... ], Luis C. López

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