Omar L. Francone

Bedford Research Foundation — United States

Medicine · Health Sciences

37h-index6.2kcitations90works0.02yr avg
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Research Topics

Cholesterol and Lipid Metabolism(35), Peroxisome Proliferator-Activated Receptors(25), Drug Transport and Resistance Mechanisms(19), Lipoproteins and Cardiovascular Health(14), Diabetes, Cardiovascular Risks, and Lipoproteins(12)

Publications90 total

Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic Leukodystrophy<i>Arsa</i>Knock-Out Mice
Journal of Neuroscience·2023· 6 citedOpen Access
Gene therapy for metachromatic leukodystrophy: Lead candidate optimization
Molecular Genetics and Metabolism·2023
Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS
Molecular Therapy — Methods & Clinical Development·2022· 5 citedOpen Access
Summary of nonclinical data for gene therapy developmental candidate HMI-203 for mucopolysaccharidosis type II (MPS II, or Hunter syndrome)
Molecular Genetics and Metabolism·2022
AAVHSCs and nervous system-targeted gene therapy for lysosomal disorders
Molecular Genetics and Metabolism·2022
Clinical trial design for HMI-203 investigational gene therapy for mucopolysaccharidosis type II (MPS II) informed by cross-correction potential and KOL input
Molecular Genetics and Metabolism·2022
AAVHSCs, a Nuclease-Independent Approach for Transduction in Non-human Primate Brain and Retina and Editing of Retinal Cells in Human Organotypic Explants
2021
HMI-203: Gene therapy developmental candidate for mucopolysaccharidosis type II (MPS II), or Hunter syndrome
Molecular Genetics and Metabolism·2021
HMI-203: Investigational gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome
Molecular Genetics and Metabolism·2021
HMI-202: A gene therapy development candidate for metachromatic leukodystrophy (MLD)
Molecular Genetics and Metabolism·2021
Molecular characterization of precise in vivo targeted gene integration in human cells using AAVHSC15
PLoS ONE·2020· 12 citedOpen Access
Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH
Molecular Therapy — Methods & Clinical Development·2020· 29 citedOpen Access
HMI-202: Investigational gene therapy for treatment of metachromatic leukodystrophy (MLD)
Molecular Genetics and Metabolism·2020
AAVHSCs target multiple cell types in the eye and have potential to treat rare retinal diseases
Investigative Ophthalmology & Visual Science·2019Open Access
Identification and functional analysis of missense mutations in the lecithin cholesterol acyltransferase gene in a Chilean patient with hypoalphalipoproteinemia
Lipids in Health and Disease·2019· 11 citedOpen Access
Single intravenous dose of AAVHSC15 vector with human phenylalanine hydroxylase transgene results in sustained correction of phenylketonuria in the PAH mouse model
Molecular Genetics and Metabolism·2019
Structure of the lipoprotein lipase–GPIHBP1 complex that mediates plasma triglyceride hydrolysis
Proceedings of the National Academy of Sciences·2018· 91 citedOpen Access
Abstract 181: The Use of Recombinant Human Lipoprotein Lipase as a Treatment for Hypertriglyceridemia
Arteriosclerosis Thrombosis and Vascular Biology·2018
A7 Drug discovery approach for rare neurological diseases: using novel zinc finger protein technology to develop potential therapy for huntington’s disease
Journal of Neurology Neurosurgery & Psychiatry·2016· 1 cited
Intravenously Administered Recombinant Human Type VII Collagen Derived from Chinese Hamster Ovary Cells Reverses the Disease Phenotype in Recessive Dystrophic Epidermolysis Bullosa Mice
Journal of Investigative Dermatology·2015· 42 citedOpen Access
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Frequent Co-authors

Lori Royer(18), Mehrdad Haghpassand(10), George H. Rothblat(8), Robert J. Aiello(6), Alan R. Tall(4), Dominique Brees(4), Edward M. Rubin(4), Christopher D. Kane(4), John McNeish(3), Charles E. Aldinger(3), Kenneth L. Hoppe(3), Patricia-Ann K. Bourassa(3), C J Fielding(3), John P. Kane(3), Christopher J. Fielding(3), Kimberly A. Stevens(3), Xian‐Cheng Jiang(2), Can Bruce(2), Anna E. Bortnick(2), Yukihiko Ueda(2)