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- [22] AAV8-Mediated Gene Therapy Rescues Retinal Degeneration Phenotype in a Tlcd3b Knockout Mouse ModelINVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2022, 63 (03)Qian, Xinye论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USA Baylor Coll Med, Human Genome Sequencing Ctr, Houston, TX USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USALiu, Hehe论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Human Genome Sequencing Ctr, Houston, TX USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USAFu, Shangyi论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Human Genome Sequencing Ctr, Houston, TX USA Baylor Coll Med, Sch Med, Houston, TX USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USALu, Jiaxiong论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Human Genome Sequencing Ctr, Houston, TX USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USAHung, Yu-Ting论文数: 0 引用数: 0 h-index: 0机构: Arizona State Univ, Coll Hlth Solut, Arizona Metab Lab, Scottsdale, AZ USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USATurner, Cassidy论文数: 0 引用数: 0 h-index: 0机构: Arizona State Univ, Coll Hlth Solut, Arizona Metab Lab, Scottsdale, AZ USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USAGu, Haiwei论文数: 0 引用数: 0 h-index: 0机构: Arizona State Univ, Coll Hlth Solut, Arizona Metab Lab, Scottsdale, AZ USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USAChen, Rui论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol & Human Genet, Houston, TX USA Baylor Coll Med, Verna & Marrs McLean Dept Biochem & Mol Biol, Houston, TX USA
- [23] Ad- and AAV8-mediated ABCA1 gene therapy in a murine model with retinal ischemia/reperfusion injuriesMOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2021, 20 : 551 - 558Luo, Jing论文数: 0 引用数: 0 h-index: 0机构: Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R China Jinan Univ, Sch Pharm, Guangzhou 510632, Peoples R China Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R ChinaWang, Shengli论文数: 0 引用数: 0 h-index: 0机构: Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R China Jinan Univ, Sch Pharm, Guangzhou 510632, Peoples R China Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R ChinaZhou, Zhenlong论文数: 0 引用数: 0 h-index: 0机构: Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R China Jinan Univ, Sch Pharm, Guangzhou 510632, Peoples R China Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R ChinaZhao, Yin论文数: 0 引用数: 0 h-index: 0机构: Huazhong Univ Sci & Technol, Tongji Hosp, Tongji Med Coll, Dept Ophthalmol, Wuhan 430030, Peoples R China Jinan Univ, Biomed Translat Res Inst, Affiliated Hosp 1, Guangzhou 510632, Peoples R China
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- [25] Prohypertrophic Agents Enhance the Response to Gene Therapy in Pompe DiseaseMOLECULAR THERAPY, 2016, 24 : S152 - S152Han, Sang-oh论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Med Ctr, Dept Pediat Med Genet, Durham, NC USA Duke Univ, Med Ctr, Dept Pediat Med Genet, Durham, NC USALi, Songtao论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Med Ctr, Dept Pediat Med Genet, Durham, NC USA Duke Univ, Med Ctr, Dept Pediat Med Genet, Durham, NC USAKoeberl, Dwight D.论文数: 0 引用数: 0 h-index: 0机构: Duke Univ, Med Ctr, Dept Pediat Med Genet, Durham, NC USA Duke Univ, Med Ctr, Dept Pediat Med Genet, Durham, NC USA
- [26] Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa)JOURNAL OF INHERITED METABOLIC DISEASE, 2025, 48 (02)Weinstein, David A.论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Dept Pediat, Farmington, CT 06269 USA Childrens Mem Hermann Hosp, Houston, TX USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USADerks, Terry G.论文数: 0 引用数: 0 h-index: 0机构: Univ Groningen, Univ Med Ctr Groningen, Beatrix Childrens Hosp, Sect Metab Dis, Groningen, Netherlands Univ Connecticut, Dept Pediat, Farmington, CT 06269 USARodriguez-Buritica, David F.论文数: 0 引用数: 0 h-index: 0机构: Univ Texas, Dept Pediat, Div Med Genet, McGovern Med Sch,Hlth Sci Ctr Houston UTHealth Hou, Houston, TX USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAAhmad, Ayesha论文数: 0 引用数: 0 h-index: 0机构: Univ Michigan, Ann Arbor, MI USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USACouce, Maria-Luz论文数: 0 引用数: 0 h-index: 0机构: Univ Clin Hosp Santiago Compostela, IDIS, CIBERER, Santiago De Compostela, Spain Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAMitchell, John J.论文数: 0 引用数: 0 h-index: 0机构: Montreal Childrens Hosp, Montreal, PQ, Canada Univ Connecticut, Dept Pediat, Farmington, CT 06269 USARiba-Wolman, Rebecca论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Dept Pediat, Farmington, CT 06269 USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAMount, Malaya论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Dept Pediat, Farmington, CT 06269 USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USASallago, Julieta Bonvin论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Dept Pediat, Farmington, CT 06269 USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USARoss, Katalin M.论文数: 0 引用数: 0 h-index: 0机构: Univ Connecticut, Dept Pediat, Farmington, CT 06269 USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAvan Der Klauw, Melanie M.论文数: 0 引用数: 0 h-index: 0机构: Univ Groningen, Univ Med Ctr Groningen, Dept Endocrinol, Groningen, Netherlands Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAde Boer, Foekje论文数: 0 引用数: 0 h-index: 0机构: Univ Groningen, Univ Med Ctr Groningen, Beatrix Childrens Hosp, Sect Metab Dis, Groningen, Netherlands Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAvan Der Schaaf, Caroline论文数: 0 引用数: 0 h-index: 0机构: Univ Groningen, Univ Med Ctr Groningen, Beatrix Childrens Hosp, Sect Metab Dis, Groningen, Netherlands Univ Connecticut, Dept Pediat, Farmington, CT 06269 USASaavedra, Heather论文数: 0 引用数: 0 h-index: 0机构: Univ Texas, Dept Pediat, Div Med Genet, McGovern Med Sch,Hlth Sci Ctr Houston UTHealth Hou, Houston, TX USA Childrens Mem Hermann Hosp, Houston, TX USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAMartinez-Olmos, Miguel论文数: 0 引用数: 0 h-index: 0机构: Univ Clin Hosp Santiago Compostela, IDIS, CIBERER, Santiago De Compostela, Spain Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAAtanga, Elvis论文数: 0 引用数: 0 h-index: 0机构: Montreal Childrens Hosp, Montreal, PQ, Canada Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAHosseini, Asad论文数: 0 引用数: 0 h-index: 0机构: Montreal Childrens Hosp, Montreal, PQ, Canada Univ Connecticut, Dept Pediat, Farmington, CT 06269 USAMitragotri, Deepali论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Pharmaceut Inc, Novato, CA USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USACrombez, Eric论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Pharmaceut Inc, Novato, CA USA Univ Connecticut, Dept Pediat, Farmington, CT 06269 USA
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