Douglas M McCarty
Overview
Explore the profile of Douglas M McCarty including associated specialties, affiliations and a list of published articles.
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33
Citations
1485
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Recent Articles
1.
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...
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Fu H, Zaraspe K, Murakami N, Meadows A, Pineda R, McCarty D, et al.
Mol Ther Methods Clin Dev
. 2018 Sep;
10:327-340.
PMID: 30191159
No treatment is available to address the neurological need and reversibility of MPS II. We developed a scAAV9-h vector to deliver the human iduronate-2-sulfatase gene and test it in mouse...
4.
Fu H, Meadows A, Pineda R, Kunkler K, Truxal K, McBride K, et al.
Hum Gene Ther Clin Dev
. 2017 Oct;
28(4):187-196.
PMID: 29064732
Recombinant adeno-associated virus (AAV) vectors are promising gene therapy tools. However, pre-existing antibodies (Abs) to many useful AAV serotypes pose a critical challenge for the translation of gene therapies. As...
5.
Fu H, Meadows A, Pineda R, Mohney R, Stirdivant S, McCarty D
Metab Brain Dis
. 2017 Apr;
32(5):1403-1415.
PMID: 28382573
The monogenic defects in specific lysosomal enzymes in mucopolysaccharidosis (MPS) III lead to lysosomal storage of glycosaminoglycans and complex CNS and somatic pathology, for which the detailed mechanisms remain unclear....
6.
Velazquez V, Meadows A, Pineda R, Camboni M, McCarty D, Fu H
Mol Ther Methods Clin Dev
. 2017 Mar;
4:159-168.
PMID: 28345001
Pre-existing antibodies (Abs) to AAV pose a critical challenge for the translation of gene therapies. No effective approach is available to overcome pre-existing Abs. Given the complexity of Ab production,...
7.
Fu H, Meadows A, Ware T, Mohney R, McCarty D
Mol Ther
. 2017 Feb;
25(3):792-802.
PMID: 28143737
Mucopolysaccharidosis (MPS) IIIB is a lysosomal storage disease with complex CNS and somatic pathology due to a deficiency in α-N-acetylglucosaminidase (NAGLU). Using global metabolic profiling by mass spectrometry targeting 361...
8.
Fu H, McCarty D
Curr Opin Virol
. 2016 Sep;
21:87-92.
PMID: 27591676
The abundant vasculature of the CNS provides a compelling route of administration for the delivery of gene therapy vectors if the limitations imposed by the blood-brain-barrier (BBB) can be overcome....
9.
Fu H, Cataldi M, Ware T, Zaraspe K, Meadows A, Murrey D, et al.
Mol Ther Methods Clin Dev
. 2016 Jun;
3:16036.
PMID: 27331076
The reversibility of neuropathic lysosomal storage diseases, including MPS IIIA, is a major goal in therapeutic development, due to typically late diagnoses and a large population of untreated patients. We...
10.
Meadows A, Duncan F, Camboni M, Waligura K, Montgomery C, Zaraspe K, et al.
Hum Gene Ther Clin Dev
. 2015 Dec;
26(4):228-42.
PMID: 26684447
No treatment is currently available for mucopolysaccharidosis (MPS) IIIB, a neuropathic lysosomal storage disease due to defect in α-N-acetylglucosaminidase (NAGLU). In preparation for a clinical trial, we performed an IND-enabling...