Biomarkers for gene therapy clinical trials of lysosomal storage disorders.

Autor:	Rossi A; Department of Translational Medicine, Section of Pediatrics, University of Naples Federico II, Naples, Italy., Malvagia S; Newborn Screening, Clinical Chemistry and Pharmacology Lab, Meyer Children's Hospital IRCCS, Florence, Italy., la Marca G; Newborn Screening, Clinical Chemistry and Pharmacology Lab, Meyer Children's Hospital IRCCS, Florence, Italy; Department of Experimental and Clinical Biomedical Sciences, University of Florence, Florence, Italy., Parenti G; Department of Translational Medicine, Section of Pediatrics, University of Naples Federico II, Naples, Italy; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy; School of Advanced Studies, Genomics and Experimental Medicine Program, University of Naples Federico II, Naples, Italy., Brunetti-Pierri N; Department of Translational Medicine, Section of Pediatrics, University of Naples Federico II, Naples, Italy; Telethon Institute of Genetics and Medicine, Pozzuoli, Italy; School of Advanced Studies, Genomics and Experimental Medicine Program, University of Naples Federico II, Naples, Italy. Electronic address: brunetti@tigem.it.
Jazyk:	angličtina
Zdroj:	Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Sep 04; Vol. 32 (9), pp. 2930-2938. Date of Electronic Publication: 2024 Jun 06.
DOI:	10.1016/j.ymthe.2024.06.003
Abstrakt:	Lysosomal storage disorders (LSDs) are multisystemic progressive disorders caused by defects in proteins involved in lysosomal function. Different gene therapy strategies are under clinical investigation in several LSDs to overcome the limitations of available treatments. However, LSDs are slowly progressive diseases that require long-term studies to establish the efficacy of experimental treatments. Biomarkers can be reliable substitutes for clinical responses and improve the efficiency of clinical trials, especially when long-term disease interventions are evaluated. In this review, we summarize both available and future biomarkers for LSDs and discuss their strengths and weaknesses. Competing Interests: Declaration of interests The authors declare no competing interests. (Copyright © 2024 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.)
Databáze:	MEDLINE
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