Towards Precision Therapies for Inherited Disorders of Neurodegeneration with Brain Iron Accumulation.

ABSTRACT:

Background

Neurodegeneration with brain iron accumulation (NBIA) disorders comprise a group of rare but devastating inherited neurological diseases with unifying features of progressive cognitive and motor decline, and increased iron deposition in the basal ganglia. Although at present there are no proven disease-modifying treatments, the severe nature of these monogenic disorders lends to consideration of personalized medicine strategies, including targeted gene therapy. In this review we summarize the progress and future direction towards precision therapies for NBIA disorders.

Methods

This review considered all relevant publications up to April 2021 using a systematic search strategy of PubMed and clinical trials databases.

Results

We review what is currently known

SUBMITTER: Spaull RVV 

PROVIDER: S-EPMC8641530 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature