Jerry F Harb
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Explore the profile of Jerry F Harb including associated specialties, affiliations and a list of published articles.
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6
Citations
5
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Recent Articles
1.
Calhoun C, Kan S, Stover A, Harb J, Monuki E, Wang R, et al.
Mol Ther Methods Clin Dev
. 2025 Jan;
32(4):101367.
PMID: 39764351
Mucopolysaccharidosis type I (MPS I) is a metabolic disorder characterized by a deficiency in α-l-iduronidase (IDUA), leading to impaired glycosaminoglycan degradation. Current approved treatments seek to restore IDUA levels via...
2.
Rha A, Christensen C, Kan S, Harb J, Andrade-Heckman P, Wang R
Stem Cell Res
. 2024 Sep;
81:103552.
PMID: 39303321
GM1 gangliosidosis (GM1) is a rare autosomal recessive neurogenerative lysosomal storage disease characterized by deficiency of beta-galactosidase (β-gal) and intralysosomal accumulation of GM1 ganglioside and other glycoconjugates. Resources for GM1...
3.
Rha A, Kan S, Andrade-Heckman P, Christensen C, Harb J, Wang R
Mol Genet Metab
. 2024 Sep;
143(1-2):108568.
PMID: 39303319
GM1 gangliosidosis is an autosomal recessive neurodegenerative lysosomal storage disease caused by pathogenic variants in the GLB1 gene, limiting the production of active lysosomal β-galactosidase. Phenotypic heterogeneity is due in...
4.
Christensen C, Kan S, Andrade-Heckman P, Rha A, Harb J, Wang R
Mol Ther Nucleic Acids
. 2024 Jul;
35(2):102220.
PMID: 38948331
Infantile-onset Pompe disease (IOPD) results from pathogenic variants in the gene, which encodes acid α-glucosidase. The correction of pathogenic variants through genome editing may be a valuable one-time therapy for...
5.
Harb J, Christensen C, Kan S, Rha A, Andrade-Heckman P, Pollard L, et al.
Mol Ther Nucleic Acids
. 2023 Sep;
34:102022.
PMID: 37727271
Free sialic acid storage disorders (FSASDs) result from pathogenic variations in the gene, which encodes the lysosomal transmembrane protein sialin. Loss or deficiency of sialin impairs FSA transport out of...
6.
Kezos J, Cabral L, Wong B, Khou B, Oh A, Harb J, et al.
J Insect Physiol
. 2017 Mar;
99:8-14.
PMID: 28285040
Insects and vertebrates have multiple major physiological systems, each species having a circulatory system, a metabolic system, and a respiratory system that enable locomotion and survival in stressful environments, among...