Gene Therapy for Neuronopathic Mucopolysaccharidoses: State of the Art.

ABSTRACT: The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme replacement therapy (ERT), do not reach important areas such as the central nervous system (CNS). The advent of newborn screening procedures represents a major step forward in early identification and treatment of individuals with MPS. However, the treatment of brain disease in neuronopathic MPS has been a major challenge to date, mainly because the blood brain barrier (BBB) prevents penetration of the brain by large molecules, including enzymes. Over the last years several novel experimental therapies for neuronopathic MPS have been investigated. Gene therapy and gene edit

SUBMITTER: de Castro MJ 

PROVIDER: S-EPMC8430935 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature