Gene Therapy
Bibliographic References tagged with Gene Therapy
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Vignal-Clermont C, Yu-Wai-Man P, Newman N, Carelli V, Moster M, Biousse V, Subramanian P, Wang AG, Donahue S, Leroy B, Sadun A, Klopstock T, Sergott R, Rebolleda Fernandez G, Chwalisz B, Banik R, Taiel M, Roux M, Sahel JA, LHON Study Group. Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.
Am J Ophthalmol. 2023;249:108–125. PMID: 36496192
Vignal-Clermont C, Yu-Wai-Man P, Newman N, Carelli V, Moster M, Biousse V, Subramanian P, Wang AG, Donahue S, Leroy B, Sadun A, Klopstock T, Sergott R, Rebolleda Fernandez G, Chwalisz B, Banik R, Taiel M, Roux M, Sahel JA, LHON Study Group. Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.
Am J Ophthalmol. 2023;249:108–125. PMID: 36496192
Girach A, Audo I, Birch D, Huckfeldt R, Lam B, Leroy B, Michaelides M, Russell S, Sallum J, Stingl K, Tsang S, Yang P. RNA-based therapies in inherited retinal diseases.
Ther Adv Ophthalmol. 2022;14:25158414221134602. PMID: 36388727
Girach A, Audo I, Birch D, Huckfeldt R, Lam B, Leroy B, Michaelides M, Russell S, Sallum J, Stingl K, Tsang S, Yang P. RNA-based therapies in inherited retinal diseases.
Ther Adv Ophthalmol. 2022;14:25158414221134602. PMID: 36388727
Peng DW, Lan CL, Dong LQ, Jiang MX, Xiao H, D’Amato R, Chi ZL. Anti-angiogenic properties of microRNA-29a in preclinical ocular models.
Proc Natl Acad Sci U S A. 2022;119(45):e2204795119. PMID: 36322719
Peng DW, Lan CL, Dong LQ, Jiang MX, Xiao H, D’Amato R, Chi ZL. Anti-angiogenic properties of microRNA-29a in preclinical ocular models.
Proc Natl Acad Sci U S A. 2022;119(45):e2204795119. PMID: 36322719
Zinn E, Unzu C, Schmit P, Turunen H, Zabaleta N, Sanmiguel J, Fieldsend A, Bhatt U, Diop C, Merkel E, Gurrala R, Peacker B, Rios C, Messemer K, Santos J, Estelien R, Andres-Mateos E, Wagers A, Tipper C, Vandenberghe L. Ancestral library identifies conserved reprogrammable liver motif on AAV capsid.
Cell Rep Med. 2022;3(11):100803. PMID: 36327973
Zinn E, Unzu C, Schmit P, Turunen H, Zabaleta N, Sanmiguel J, Fieldsend A, Bhatt U, Diop C, Merkel E, Gurrala R, Peacker B, Rios C, Messemer K, Santos J, Estelien R, Andres-Mateos E, Wagers A, Tipper C, Vandenberghe L. Ancestral library identifies conserved reprogrammable liver motif on AAV capsid.
Cell Rep Med. 2022;3(11):100803. PMID: 36327973
Newman N, Yu-Wai-Man P, Subramanian P, Moster M, Wang AG, Donahue S, Leroy B, Carelli V, Biousse V, Vignal-Clermont C, Sergott R, Sadun A, Fernández GR, Chwalisz B, Banik R, Bazin F, Roux M, Cox E, Taiel M, Sahel JA, LHON REFLECT Study Group. Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Brain. 2023;146(4):1328–1341. PMID: 36350566
Newman N, Yu-Wai-Man P, Subramanian P, Moster M, Wang AG, Donahue S, Leroy B, Carelli V, Biousse V, Vignal-Clermont C, Sergott R, Sadun A, Fernández GR, Chwalisz B, Banik R, Bazin F, Roux M, Cox E, Taiel M, Sahel JA, LHON REFLECT Study Group. Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Brain. 2023;146(4):1328–1341. PMID: 36350566
Sharma J, Paschalis E. The future of non-viral gene delivery for the treatment of inherited retinal diseases.
Mol Ther Nucleic Acids. 2022;30:354. PMID: 36381582
Sharma J, Paschalis E. The future of non-viral gene delivery for the treatment of inherited retinal diseases.
Mol Ther Nucleic Acids. 2022;30:354. PMID: 36381582
Whitman M, Gilette N, Bell J, Kim S, Tischfield M, Engle E. TWIST1, a gene associated with Saethre-Chotzen syndrome, regulates extraocular muscle organization in mouse.
Dev Biol. 2022;490:126–133. PMID: 35944701
Whitman M, Gilette N, Bell J, Kim S, Tischfield M, Engle E. TWIST1, a gene associated with Saethre-Chotzen syndrome, regulates extraocular muscle organization in mouse.
Dev Biol. 2022;490:126–133. PMID: 35944701
Lu YC, Tsai YH, Chan YH, Hu CJ, Huang CY, Xiao R, Hsu CJ, Vandenberghe L, Wu CC, Cheng YF. Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice.
JCI Insight. 2022;7(20). PMID: 36278489
Lu YC, Tsai YH, Chan YH, Hu CJ, Huang CY, Xiao R, Hsu CJ, Vandenberghe L, Wu CC, Cheng YF. Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice.
JCI Insight. 2022;7(20). PMID: 36278489
Kuo A, Checa A, Niaudet C, Jung B, Fu Z, Wheelock C, Singh S, Aikawa M, Smith L, Proia R, Hla T. Murine endothelial serine palmitoyltransferase 1 (SPTLC1) is required for vascular development and systemic sphingolipid homeostasis.
Elife. 2022;11. PMID: 36197001
Kuo A, Checa A, Niaudet C, Jung B, Fu Z, Wheelock C, Singh S, Aikawa M, Smith L, Proia R, Hla T. Murine endothelial serine palmitoyltransferase 1 (SPTLC1) is required for vascular development and systemic sphingolipid homeostasis.
Elife. 2022;11. PMID: 36197001
Whitman M, Gilette N, Bell J, Kim S, Tischfield, Engle E. TWIST1, a gene associated with Saethre-Chotzen syndrome, regulates extraocular muscleorganization in mouse.
Dev Biol. 2022;
Whitman M, Gilette N, Bell J, Kim S, Tischfield, Engle E. TWIST1, a gene associated with Saethre-Chotzen syndrome, regulates extraocular muscleorganization in mouse.
Dev Biol. 2022;