Chandler RJ - Search Results

1

Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience.

Lomash RM, Shchelochkov O, Chandler RJ, Venditti CP, Pariser AR, Ottinger EA; NIH PaVe-GT Team. Lomash RM, et al. Among authors: chandler rj. Hum Gene Ther. 2023 Mar;34(5-6):217-227. doi: 10.1089/hum.2022.232. Hum Gene Ther. 2023. PMID: 36694456 Free PMC article.

2

Genetic and genomic systems to study methylmalonic acidemia.

Chandler RJ, Venditti CP. Chandler RJ, et al. Mol Genet Metab. 2005 Sep-Oct;86(1-2):34-43. doi: 10.1016/j.ymgme.2005.07.020. Epub 2005 Sep 22. Mol Genet Metab. 2005. PMID: 16182581 Free PMC article. Review.

3

Propionyl-CoA and adenosylcobalamin metabolism in Caenorhabditis elegans: evidence for a role of methylmalonyl-CoA epimerase in intermediary metabolism.

Chandler RJ, Aswani V, Tsai MS, Falk M, Wehrli N, Stabler S, Allen R, Sedensky M, Kazazian HH, Venditti CP. Chandler RJ, et al. Mol Genet Metab. 2006 Sep-Oct;89(1-2):64-73. doi: 10.1016/j.ymgme.2006.06.001. Epub 2006 Jul 14. Mol Genet Metab. 2006. PMID: 16843692 Free PMC article.

4

Adenoviral-mediated correction of methylmalonyl-CoA mutase deficiency in murine fibroblasts and human hepatocytes.

Chandler RJ, Tsai MS, Dorko K, Sloan J, Korson M, Freeman R, Strom S, Venditti CP. Chandler RJ, et al. BMC Med Genet. 2007 Apr 30;8:24. doi: 10.1186/1471-2350-8-24. BMC Med Genet. 2007. PMID: 17470278 Free PMC article.

5

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. Chandler RJ, et al. BMC Med Genet. 2007 Oct 15;8:64. doi: 10.1186/1471-2350-8-64. BMC Med Genet. 2007. PMID: 17937813 Free PMC article.

6

Adenovirus-mediated gene delivery rescues a neonatal lethal murine model of mut(0) methylmalonic acidemia.

Chandler RJ, Venditti CP. Chandler RJ, et al. Hum Gene Ther. 2008 Jan;19(1):53-60. doi: 10.1089/hum.2007.0118. Hum Gene Ther. 2008. PMID: 18052792 Free PMC article.

7

Mitochondrial dysfunction in mut methylmalonic acidemia.

Chandler RJ, Zerfas PM, Shanske S, Sloan J, Hoffmann V, DiMauro S, Venditti CP. Chandler RJ, et al. FASEB J. 2009 Apr;23(4):1252-61. doi: 10.1096/fj.08-121848. Epub 2008 Dec 16. FASEB J. 2009. PMID: 19088183 Free PMC article.

8

Long-term rescue of a lethal murine model of methylmalonic acidemia using adeno-associated viral gene therapy.

Chandler RJ, Venditti CP. Chandler RJ, et al. Mol Ther. 2010 Jan;18(1):11-6. doi: 10.1038/mt.2009.247. Epub 2009 Oct 27. Mol Ther. 2010. PMID: 19861951 Free PMC article.

9

Ion-abrasion scanning electron microscopy reveals distorted liver mitochondrial morphology in murine methylmalonic acidemia.

Murphy GE, Lowekamp BC, Zerfas PM, Chandler RJ, Narasimha R, Venditti CP, Subramaniam S. Murphy GE, et al. Among authors: chandler rj. J Struct Biol. 2010 Aug;171(2):125-32. doi: 10.1016/j.jsb.2010.04.005. Epub 2010 Apr 23. J Struct Biol. 2010. PMID: 20399866 Free PMC article.

10

Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction.

Carrillo-Carrasco N, Chandler RJ, Chandrasekaran S, Venditti CP. Carrillo-Carrasco N, et al. Among authors: chandler rj. Hum Gene Ther. 2010 Sep;21(9):1147-54. doi: 10.1089/hum.2010.008. Hum Gene Ther. 2010. PMID: 20486773 Free PMC article.

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