Randy J Chandler
Overview
Explore the profile of Randy J Chandler including associated specialties, affiliations and a list of published articles.
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Articles
44
Citations
1324
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Recent Articles
1.
Lomash R, Dehdashti J, Shchelochkov O, Chandler R, Li L, Manoli I, et al.
Hum Gene Ther
. 2025 Feb;
36(5-6):653-662.
PMID: 39976996
Gene therapy development presents multiple challenges, and early planning is vital in the successful implementation of such programs. The Platform Vector Gene Therapy (PaVe-GT) program is a National Institutes of...
2.
Chandler R
Mol Ther Methods Clin Dev
. 2024 Dec;
32(4):101373.
PMID: 39649706
No abstract available.
3.
Romero D, Chandler R
J Vis Exp
. 2024 Jun;
(207).
PMID: 38829107
While tail vein injections are frequently used as a systemic route of delivery in adult mice, retrobulbar injections are an alternative method for systemic delivery with fewer limitations. First, tail...
4.
Chandler R, Di Pasquale G, Choi E, Chang D, Smith S, Sloan J, et al.
Mol Ther Methods Clin Dev
. 2023 Sep;
30:181-190.
PMID: 37746248
Propionic acidemia (PA) is rare autosomal recessive metabolic disorder caused by defects in the mitochondrially localized enzyme propionyl-coenzyme A (CoA) carboxylase. Patients with PA can suffer from lethal metabolic decompensation...
5.
Chandler R, Venditti C
J Inherit Metab Dis
. 2023 Aug;
47(1):63-79.
PMID: 37530705
Organic acidemias (OA) are a group of rare autosomal recessive disorders of intermediary metabolism that result in a systemic elevation of organic acid. Despite optimal dietary and cofactor therapy, OA...
6.
From Puppies to adults: editing of hepatocytes in a canine model of glycogen storage disease type Ia
Chandler R
Mol Ther Methods Clin Dev
. 2023 May;
29:347-349.
PMID: 37206367
No abstract available.
7.
Lomash R, Shchelochkov O, Chandler R, Venditti C, Pariser A, Ottinger E
Hum Gene Ther
. 2023 Jan;
34(5-6):217-227.
PMID: 36694456
Orphan drug designation (ODD) is an important program intended to facilitate the development of orphan drugs in the United States. An orphan drug benefiting pediatric patients can qualify as a...
8.
Chandler R, Di Pasquale G, Sloan J, McCoy S, Hubbard B, Kilts T, et al.
Mol Ther Methods Clin Dev
. 2022 Oct;
27:61-72.
PMID: 36186952
Methylmalonic acidemia (MMA) is a severe and potentially lethal autosomal recessive inborn error of metabolism most frequently caused by mutations in the methylmalonyl-CoA mutase () gene. Proof-of-concept adeno-associated virus (AAV)...
9.
Venturoni L, Chandler R, Liao J, Hoffmann V, Ramesh N, Gordo S, et al.
Mol Genet Metab
. 2022 Jul;
137(1-2):1-8.
PMID: 35868241
Methylmalonic acidemia (MMA) is a rare and severe inherited metabolic disease typically caused by mutations of the methylmalonyl-CoA mutase (MMUT) gene. Despite medical management, patients with MMA experience frequent episodes...
10.
Sabatino D, Bushman F, Chandler R, Crystal R, Davidson B, Dolmetsch R, et al.
Mol Ther
. 2022 Jun;
30(8):2646-2663.
PMID: 35690906
On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in preclinical and clinical AAV...