189 related articles for article (PubMed ID: 34169115)
1. Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia.
May FJ; Head PE; Venturoni LE; Chandler RJ; Venditti CP
Mol Ther Methods Clin Dev; 2021 Jun; 21():765-776. PubMed ID: 34169115
[TBL] [Abstract][Full Text] [Related]
2. Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing.
Venturoni LE; Chandler RJ; Liao J; Hoffmann V; Ramesh N; Gordo S; Chau N; Venditti CP
Mol Genet Metab; 2022; 137(1-2):1-8. PubMed ID: 35868241
[TBL] [Abstract][Full Text] [Related]
3. Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.
Chandler RJ; Venturoni LE; Liao J; Hubbard BT; Schneller JL; Hoffmann V; Gordo S; Zang S; Ko CW; Chau N; Chiang K; Kay MA; Barzel A; Venditti CP
Hepatology; 2021 Jun; 73(6):2223-2237. PubMed ID: 32976669
[TBL] [Abstract][Full Text] [Related]
4. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9.
Chandler RJ; Di Pasquale G; Sloan JL; McCoy S; Hubbard BT; Kilts TM; Manoli I; Chiorini JA; Venditti CP
Mol Ther Methods Clin Dev; 2022 Dec; 27():61-72. PubMed ID: 36186952
[TBL] [Abstract][Full Text] [Related]
5.
Schneller JL; Lee CM; Venturoni LE; Chandler RJ; Li A; Myung S; Cradick TJ; Hurley AE; Lagor WR; Bao G; Venditti CP
Mol Ther Methods Clin Dev; 2021 Dec; 23():619-632. PubMed ID: 34901307
[TBL] [Abstract][Full Text] [Related]
6. Treatment of metabolic disorders using genomic technologies: Lessons from methylmalonic acidemia.
Venturoni LE; Venditti CP
J Inherit Metab Dis; 2022 Sep; 45(5):872-888. PubMed ID: 35766386
[TBL] [Abstract][Full Text] [Related]
7. Novel AAV-mediated genome editing therapy improves health and survival in a mouse model of methylmalonic acidemia.
Zhang S; Bastille A; Gordo S; Ramesh N; Vora J; McCarthy E; Zhang X; Frank D; Ko CW; Wu C; Walsh N; Amarwani S; Liao J; Xiong Q; Drouin L; Hebben M; Chiang K; Chau BN
PLoS One; 2022; 17(9):e0274774. PubMed ID: 36126056
[TBL] [Abstract][Full Text] [Related]
8. A study on a cohort of 301 Chinese patients with isolated methylmalonic acidemia.
Kang L; Liu Y; Shen M; Liu Y; He R; Song J; Jin Y; Li M; Zhang Y; Dong H; Liu X; Yan H; Qin J; Zheng H; Chen Y; Li D; Wei H; Zhang H; Sun L; Zhu Z; Liang D; Yang Y
J Inherit Metab Dis; 2020 May; 43(3):409-423. PubMed ID: 31622506
[TBL] [Abstract][Full Text] [Related]
9. Metabolic phenotype of methylmalonic acidemia in mice and humans: the role of skeletal muscle.
Chandler RJ; Sloan J; Fu H; Tsai M; Stabler S; Allen R; Kaestner KH; Kazazian HH; Venditti CP
BMC Med Genet; 2007 Oct; 8():64. PubMed ID: 17937813
[TBL] [Abstract][Full Text] [Related]
10. Impaired Function of a Rare Mutation in the
Dai S; Yang Y; Li Y; Liu H
Genet Res (Camb); 2022; 2022():5611697. PubMed ID: 35919035
[TBL] [Abstract][Full Text] [Related]
11. Tricarboxylic acid cycle enzyme activities in a mouse model of methylmalonic aciduria.
Wongkittichote P; Cunningham G; Summar ML; Pumbo E; Forny P; Baumgartner MR; Chapman KA
Mol Genet Metab; 2019 Dec; 128(4):444-451. PubMed ID: 31648943
[TBL] [Abstract][Full Text] [Related]
12. A novel
Zhang X; Xu X; Shu J; Zhi X; Wang H; Dong Y; Sheng W; Li D; Meng Y; Cai C
Heliyon; 2024 Mar; 10(5):e26912. PubMed ID: 38455531
[TBL] [Abstract][Full Text] [Related]
13. ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia.
Ilyinskii PO; Michaud AM; Rizzo GL; Roy CJ; Leung SS; Elkins SL; Capela T; Chowdhury A; Li L; Chandler RJ; Manoli I; Andres-Mateos E; Johnston LPM; Vandenberghe LH; Venditti CP; Kishimoto TK
Mol Ther Methods Clin Dev; 2021 Sep; 22():279-292. PubMed ID: 34485611
[TBL] [Abstract][Full Text] [Related]
14. Biochemical and anaplerotic applications of in vitro models of propionic acidemia and methylmalonic acidemia using patient-derived primary hepatocytes.
Collado MS; Armstrong AJ; Olson M; Hoang SA; Day N; Summar M; Chapman KA; Reardon J; Figler RA; Wamhoff BR
Mol Genet Metab; 2020 Jul; 130(3):183-196. PubMed ID: 32451238
[TBL] [Abstract][Full Text] [Related]
15. Biomarkers to predict disease progression and therapeutic response in isolated methylmalonic acidemia.
Manoli I; Gebremariam A; McCoy S; Pass AR; Gagné J; Hall C; Ferry S; Van Ryzin C; Sloan JL; Sacchetti E; Catesini G; Rizzo C; Martinelli D; Spada M; Dionisi-Vici C; Venditti CP
J Inherit Metab Dis; 2023 Jul; 46(4):554-572. PubMed ID: 37243446
[TBL] [Abstract][Full Text] [Related]
16. Methylmalonyl acidemia: from mitochondrial metabolism to defective mitophagy and disease.
Luciani A; Devuyst O
Autophagy; 2020 Jun; 16(6):1159-1161. PubMed ID: 32316822
[TBL] [Abstract][Full Text] [Related]
17. Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy.
Chandler RJ; Venditti CP
Mol Ther; 2010 Jan; 18(1):11-6. PubMed ID: 19861951
[TBL] [Abstract][Full Text] [Related]
18. Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery.
Sénac JS; Chandler RJ; Sysol JR; Li L; Venditti CP
Gene Ther; 2012 Apr; 19(4):385-91. PubMed ID: 21776024
[TBL] [Abstract][Full Text] [Related]
19. A novel small molecule approach for the treatment of propionic and methylmalonic acidemias.
Armstrong AJ; Collado MS; Henke BR; Olson MW; Hoang SA; Hamilton CA; Pourtaheri TD; Chapman KA; Summar MM; Johns BA; Wamhoff BR; Reardon JE; Figler RA
Mol Genet Metab; 2021 May; 133(1):71-82. PubMed ID: 33741272
[TBL] [Abstract][Full Text] [Related]
20.
; ; . PubMed ID:
[No Abstract] [Full Text] [Related]
[Next] [New Search]