Gene Therapy
Bibliographic References tagged with Gene Therapy
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Aleman T, Huckfeldt R, Serrano L, Pearson D, Vergilio G, McCague S, Marshall K, Ashtari M, Doan T, Weigel-DiFranco C, Biron B, Wen XH, Chung D, Liu E, Ferenchak K, Morgan J, Pierce E, Eliott D, Bennett J, Comander J, Maguire A. Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial.
Ophthalmology. 2022;129(10):1177–1191. PMID: 35714735
Aleman T, Huckfeldt R, Serrano L, Pearson D, Vergilio G, McCague S, Marshall K, Ashtari M, Doan T, Weigel-DiFranco C, Biron B, Wen XH, Chung D, Liu E, Ferenchak K, Morgan J, Pierce E, Eliott D, Bennett J, Comander J, Maguire A. Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial.
Ophthalmology. 2022;129(10):1177–1191. PMID: 35714735
Salamzade R, Manson A, Walker B, Brennan-Krohn T, Worby C, Ma P, He L, Shea T, Qu J, Chapman S, Howe W, Young S, Wurster J, Delaney M, Kanjilal S, Onderdonk A, Bittencourt C, Gussin G, Kim D, Peterson E, Ferraro MJ, Hooper D, Shenoy E, Cuomo C, Cosimi L, Huang S, Kirby J, Pierce V, Bhattacharyya R, Earl A. Inter-species geographic signatures for tracing horizontal gene transfer and long-term persistence of carbapenem resistance.
Genome Med. 2022;14(1):37. PMID: 35379360
Salamzade R, Manson A, Walker B, Brennan-Krohn T, Worby C, Ma P, He L, Shea T, Qu J, Chapman S, Howe W, Young S, Wurster J, Delaney M, Kanjilal S, Onderdonk A, Bittencourt C, Gussin G, Kim D, Peterson E, Ferraro MJ, Hooper D, Shenoy E, Cuomo C, Cosimi L, Huang S, Kirby J, Pierce V, Bhattacharyya R, Earl A. Inter-species geographic signatures for tracing horizontal gene transfer and long-term persistence of carbapenem resistance.
Genome Med. 2022;14(1):37. PMID: 35379360
Andres-Mateos E, Landegger L, Unzu C, Phillips J, Lin B, Dewyer N, Sanmiguel J, Nicolaou F, Valero M, Bourdeu K, Sewell W, Beiler R, McKenna M, Stankovic K, Vandenberghe L. Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate.
Nat Commun. 2022;13(1):1359. PMID: 35292639
Andres-Mateos E, Landegger L, Unzu C, Phillips J, Lin B, Dewyer N, Sanmiguel J, Nicolaou F, Valero M, Bourdeu K, Sewell W, Beiler R, McKenna M, Stankovic K, Vandenberghe L. Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate.
Nat Commun. 2022;13(1):1359. PMID: 35292639
Grotz S, Schäfer J, Wunderlich K, Ellederova Z, Auch H, Bähr A, Runa-Vochozkova P, Fadl J, Arnold V, Ardan T, Veith M, Santamaria G, Dhom G, Hitzl W, Kessler B, Eckardt C, Klein J, Brymova A, Linnert J, Kurome M, Zakharchenko V, Fischer A, Blutke A, Döring A, Suchankova S, Popelar J, Rodríguez-Bocanegra E, Dlugaiczyk J, Straka H, May-Simera H, Wang W, Laugwitz KL, Vandenberghe L, Wolf E, Nagel-Wolfrum K, Peters T, Motlik J, Fischer D, Wolfrum U, Klymiuk N. Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes.
EMBO Mol Med. 2022;14(4):e14817. PMID: 35254721
Grotz S, Schäfer J, Wunderlich K, Ellederova Z, Auch H, Bähr A, Runa-Vochozkova P, Fadl J, Arnold V, Ardan T, Veith M, Santamaria G, Dhom G, Hitzl W, Kessler B, Eckardt C, Klein J, Brymova A, Linnert J, Kurome M, Zakharchenko V, Fischer A, Blutke A, Döring A, Suchankova S, Popelar J, Rodríguez-Bocanegra E, Dlugaiczyk J, Straka H, May-Simera H, Wang W, Laugwitz KL, Vandenberghe L, Wolf E, Nagel-Wolfrum K, Peters T, Motlik J, Fischer D, Wolfrum U, Klymiuk N. Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes.
EMBO Mol Med. 2022;14(4):e14817. PMID: 35254721
Wang J, Zhao L, Gu X, Xue Y, Wang S, Xiao R, Vandenberghe L, Peng K, Shu Y, Li H. Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig.
Hum Gene Ther. 2022;33(13-14):719–728. PMID: 35156857
Wang J, Zhao L, Gu X, Xue Y, Wang S, Xiao R, Vandenberghe L, Peng K, Shu Y, Li H. Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig.
Hum Gene Ther. 2022;33(13-14):719–728. PMID: 35156857
Sobrin L, Yang J. Not All Genes Are Created Equal in Age-Related Macular Degeneration.
JAMA Ophthalmol. 2022;140(3):260–261. PMID: 35113138
Sobrin L, Yang J. Not All Genes Are Created Equal in Age-Related Macular Degeneration.
JAMA Ophthalmol. 2022;140(3):260–261. PMID: 35113138
Banskota S, Raguram A, Suh S, Du S, Davis J, Choi E, Wang X, Nielsen S, Newby G, Randolph P, Osborn M, Musunuru K, Palczewski K, Liu D. Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins.
Cell. 2022;185(2):250–265.e16. PMID: 35021064
Banskota S, Raguram A, Suh S, Du S, Davis J, Choi E, Wang X, Nielsen S, Newby G, Randolph P, Osborn M, Musunuru K, Palczewski K, Liu D. Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins.
Cell. 2022;185(2):250–265.e16. PMID: 35021064
Zolotukhin, Vandenberghe. AAV capsid design: A Goldilocks challenge.
Trends Mol Med. 2022;28(3):183–193. PMID: 35093287
Zolotukhin, Vandenberghe. AAV capsid design: A Goldilocks challenge.
Trends Mol Med. 2022;28(3):183–193. PMID: 35093287
Ferenchak K, Deitch I, Huckfeldt R. Antisense Oligonucleotide Therapy for Ophthalmic Conditions.
Semin Ophthalmol. 2021;36(5-6):452–457. PMID: 34010086
Ferenchak K, Deitch I, Huckfeldt R. Antisense Oligonucleotide Therapy for Ophthalmic Conditions.
Semin Ophthalmol. 2021;36(5-6):452–457. PMID: 34010086
Keffeler E, Iyer V, Parthasarathy S, Ramsey M, Gorman M, Barke T, Varahan S, Olson S, Gilmore M, Abdullahi Z, Hancock E, Hancock L. Influence of the Alternative Sigma Factor RpoN on Global Gene Expression and Carbon Catabolism in Enterococcus faecalis V583.
mBio. 2021;12(3). PMID: 34006651
Keffeler E, Iyer V, Parthasarathy S, Ramsey M, Gorman M, Barke T, Varahan S, Olson S, Gilmore M, Abdullahi Z, Hancock E, Hancock L. Influence of the Alternative Sigma Factor RpoN on Global Gene Expression and Carbon Catabolism in Enterococcus faecalis V583.
mBio. 2021;12(3). PMID: 34006651