Enzyme replacement therapy (ERT) and allogeneic hematopoietic stem cell transplantation (HSCT) are standard treatments for some mucopolysaccharidoses. Nevertheless, ERT is not curative, and HSCT is associated with significant mortality and morbidity, leaving a substantial disease burden of brain and skeletal manifestations. To overcome these limitations, different gene therapy (GT) strategies are under preclinical and clinical development. Data from ex-vivo GT clinical trials have demonstrated encouraging biochemical and early clinical outcomes. In-vivo GT, based on local brain delivery or systemic intravenous injections, resulted in biochemical and clinical stabilization of the disease.
Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art / Consiglieri, Giulia; Bernardo, Maria Ester; Brunetti-Pierri, Nicola; Aiuti, Alessandro. - In: HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA. - ISSN 0889-8588. - 36:4(2022), pp. 865-878. [10.1016/j.hoc.2022.03.012]
Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art
Brunetti-Pierri, Nicola;
2022
Abstract
Enzyme replacement therapy (ERT) and allogeneic hematopoietic stem cell transplantation (HSCT) are standard treatments for some mucopolysaccharidoses. Nevertheless, ERT is not curative, and HSCT is associated with significant mortality and morbidity, leaving a substantial disease burden of brain and skeletal manifestations. To overcome these limitations, different gene therapy (GT) strategies are under preclinical and clinical development. Data from ex-vivo GT clinical trials have demonstrated encouraging biochemical and early clinical outcomes. In-vivo GT, based on local brain delivery or systemic intravenous injections, resulted in biochemical and clinical stabilization of the disease.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
