Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review.

Stargardt Disease / genetics Humans Genetic Therapy / methods ATP-Binding Cassette Transporters / genetics Mutation Stem Cell Transplantation / methods Animals Macular Degeneration / therapy Retinal Pigment Epithelium / metabolism CRISPR-Cas Systems
ABCA4 gene CRISPR/Cas9 Stargardt disease gene therapy pharmacological interventions precision medicine stem cell therapy

Journal

International journal of molecular sciences
ISSN: 1422-0067
Titre abrégé: Int J Mol Sci
Pays: Switzerland
ID NLM: 101092791

Informations de publication

Date de publication:
14 Aug 2024
Historique:
received: 21 07 2024
revised: 09 08 2024
accepted: 12 08 2024
medline: 31 8 2024
pubmed: 31 8 2024
entrez: 29 8 2024
Statut: epublish

Résumé

Stargardt disease, one of the most common forms of inherited retinal diseases, affects individuals worldwide. The primary cause is mutations in the

Identifiants

DOI: 10.3390/ijms25168859 PMID: 39201545
pubmed: 39201545
pii: ijms25168859
doi: 10.3390/ijms25168859
pii:
doi:

Substances chimiques

ABCA4 protein, human 0
ATP-Binding Cassette Transporters 0

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Auteurs

Laura Andreea Ghenciu (LA)

Department of Functional Sciences, "Victor Babeș" University of Medicine and Pharmacy Timișoara, Eftimie Murgu Square No. 2, 300041 Timișoara, Romania.
ORCID: 0000-0002-5326-5438

Ovidiu Alin Hațegan (OA)

Discipline of Anatomy and Embriology, Medicine Faculty, Vasile Goldis Western University of Arad, Revolution Boulevard 94, 310025 Arad, Romania.

Emil Robert Stoicescu (ER)

Field of Applied Engineering Sciences, Specialization Statistical Methods and Techniques in Health and Clinical Research, Faculty of Mechanics, 'Politehnica' University Timișoara, Mihai Viteazul Boulevard No. 1, 300222 Timișoara, Romania.
Department of Radiology and Medical Imaging, 'Victor Babeș' University of Medicine and Pharmacy Timișoara, Eftimie Murgu Square No. 2, 300041 Timișoara, Romania.
Research Center for Pharmaco-Toxicological Evaluations, 'Victor Babeș' University of Medicine and Pharmacy Timișoara, Eftimie Murgu Square No. 2, 300041 Timișoara, Romania.
ORCID: 0000-0003-4620-3340

Roxana Iacob (R)

Field of Applied Engineering Sciences, Specialization Statistical Methods and Techniques in Health and Clinical Research, Faculty of Mechanics, 'Politehnica' University Timișoara, Mihai Viteazul Boulevard No. 1, 300222 Timișoara, Romania.
Department of Anatomy and Embriology, 'Victor Babeș' University of Medicine and Pharmacy Timișoara, 300041 Timișoara, Romania.
ORCID: 0000-0002-2894-712X

Alina Maria Șișu (AM)

Department of Anatomy and Embriology, 'Victor Babeș' University of Medicine and Pharmacy Timișoara, 300041 Timișoara, Romania.

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Classifications MeSH

questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Maladies héréditaires de l'oeil › Maladie de Stargardt › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines membranaires › Protéines de transport membranaire › Transporteurs ABC › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Phénomènes génétiques › Variation génétique › Mutation › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Procédures de chirurgie opératoire › Transplantation › Transplantation cellulaire › Transplantation de cellules souches › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Eucaryotes › Animaux › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Maladies de l'oeil › Rétinopathies › Dégénérescence de la rétine › Dégénérescence maculaire › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Organes des sens › Oeil › Rétine › Épithélium pigmentaire de la rétine › Emerging Therapeutic Approaches and Genetic...
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Systèmes CRISPR-Cas › Emerging Therapeutic Approaches and Genetic...