Ronald G Crystal
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Explore the profile of Ronald G Crystal including associated specialties, affiliations and a list of published articles.
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354
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12421
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Recent Articles
1.
Sondhi D, Kaminsky S, Rosenberg J, Rostami M, Hackett N, Crystal R
Hum Gene Ther
. 2025 Jan;
36(1-2):28-35.
PMID: 39745261
CLN2 disease (late infantile neuronal ceroid lipofuscinosis) is an autosomal recessive, neurodegenerative lysosomal storage disease that results from loss of function mutations in the gene, which encodes tripeptidyl peptidase 1....
2.
Gioulvanidou M, Sarklioglu S, Chen X, Lebedeva I, Inalman Y, Pohl M, et al.
Hum Gene Ther
. 2024 Dec;
36(1-2):11-27.
PMID: 39725494
Chronic hypereosinophilia, defined as persistent elevated blood levels of eosinophils ≥1,500/μL, is associated with tissue infiltration of eosinophils and consequent organ damage by eosinophil release of toxic mediators. The current...
3.
Gunaydin C, Sondhi D, Kaminsky S, Lephart H, Leopold P, Hackett N, et al.
Mol Ther
. 2024 Nov;
32(12):4303-4318.
PMID: 39511891
Gene therapy to treat hereditary disorders conventionally delivers the normal allele to compensate for loss-of-function mutations. More effective gene therapy may be achieved using a gain-of-function variant. We tested the...
4.
Karan K, Andrzejewski S, Stiles K, Hackett N, Crystal R
Hum Gene Ther
. 2024 Sep;
35(21-22):904-916.
PMID: 39318239
The homozygous Apolipoprotein E (APOE4) genotype is the major risk factor for the development of early Alzheimer's disease. Genome engineering studies in mouse models of human APOE4-dependent pathology have established...
5.
Nakamura K, Henry T, Traverse J, Latter D, Mokadam N, Answini G, et al.
Circ Cardiovasc Interv
. 2024 May;
17(5):e014054.
PMID: 38696284
Background: XC001 is a novel adenoviral-5 vector designed to express multiple isoforms of VEGF (vascular endothelial growth factor) and more safely and potently induce angiogenesis. The EXACT trial (Epicardial Delivery...
6.
Zaidman C, Goedeker N, Aqul A, Butterfield R, Connolly A, Crystal R, et al.
J Neuromuscul Dis
. 2024 Apr;
11(3):687-699.
PMID: 38607761
Background: Duchenne muscular dystrophy (DMD) is a rare, degenerative, recessive X-linked neuromuscular disease. Mutations in the gene encoding dystrophin lead to the absence of functional dystrophin protein. Individuals living with...
7.
Rojsajjakul T, Selvan N, De B, Rosenberg J, Kaminsky S, Sondhi D, et al.
Sci Rep
. 2024 Apr;
14(1):8391.
PMID: 38600238
Friedreich's ataxia is a degenerative and progressive multisystem disorder caused by mutations in the highly conserved frataxin (FXN) gene that results in FXN protein deficiency and mitochondrial dysfunction. While gene...
8.
Rojsajjakul T, Selvan N, De B, Rosenberg J, Kaminsky S, Sondhi D, et al.
Res Sq
. 2024 Jan;
PMID: 38234818
Friedreich's ataxia is a degenerative and progressive multisystem disorder caused by mutations in the highly conserved frataxin (FXN) gene that results in FXN protein deficiency and mitochondrial dysfunction. While gene...
9.
Rostami M, Leopold P, Vasquez J, de Mulder Rougvie M, Al Shakaki A, Ait Hssain A, et al.
Mol Ther Methods Clin Dev
. 2023 Dec;
31:101136.
PMID: 38089635
Based on the observation that humans have variable responses of gene expression with the same dose of an adeno-associated vector, we hypothesized that there are deleterious variants in genes coding...
10.
Crystal R
Am J Respir Crit Care Med
. 2023 Dec;
209(2):123-124.
PMID: 38051107
No abstract available.