Inborn errors of metabolism include several genetic disorders due to disruption of cellular biochemical reactions. Although individually rare, collectively they are a large and heterogenous group of diseases affecting a significant proportion of patients. Available treatments are often unsatisfactory. Liver-directed gene therapy has potential for treatment of several inborn errors of metabolism. Although lentiviral vectors and lipid nanoparticle-mRNA have shown attractive features in preclinical studies and still have to be investigated in humans, adeno-associated virus (AAV) vectors have shown clinical success in both preclinical and clinical trials for in vivo liver-directed gene therapy. Moreover, AAV vectors have been used for genome editing.
Liver-directed gene-based therapies for inborn errors of metabolism / Piccolo, Pasquale; Rossi, Alessandro; Brunetti-Pierri, Nicola. - In: EXPERT OPINION ON BIOLOGICAL THERAPY. - ISSN 1471-2598. - 21:2(2021), pp. 229-240. [10.1080/14712598.2020.1817375]
Liver-directed gene-based therapies for inborn errors of metabolism
Rossi, Alessandro;Brunetti-Pierri, Nicola
2021
Abstract
Inborn errors of metabolism include several genetic disorders due to disruption of cellular biochemical reactions. Although individually rare, collectively they are a large and heterogenous group of diseases affecting a significant proportion of patients. Available treatments are often unsatisfactory. Liver-directed gene therapy has potential for treatment of several inborn errors of metabolism. Although lentiviral vectors and lipid nanoparticle-mRNA have shown attractive features in preclinical studies and still have to be investigated in humans, adeno-associated virus (AAV) vectors have shown clinical success in both preclinical and clinical trials for in vivo liver-directed gene therapy. Moreover, AAV vectors have been used for genome editing.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.