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.
Biomarkers for gene therapy clinical trials of lysosomal storage disorders / Rossi, Alessandro; Malvagia, Sabrina; la Marca, Giancarlo; Parenti, Giancarlo; Brunetti-Pierri, Nicola. - In: MOLECULAR THERAPY. - ISSN 1525-0024. - ELETTRONICO. - 32:(2024), pp. 0-0. [10.1016/j.ymthe.2024.06.003]
Biomarkers for gene therapy clinical trials of lysosomal storage disorders
la Marca, Giancarlo;
2024
Abstract
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.
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