Gene therapy for primary mitochondrial diseases: experimental advances and clinical challenges.
Nat Rev Neurol
; 18(11): 689-698, 2022 11.
Article
em En
| MEDLINE
| ID: mdl-36257993
ABSTRACT
The variable clinical and biochemical manifestations of primary mitochondrial diseases (PMDs), and the complexity of mitochondrial genetics, have proven to be a substantial barrier to the development of effective disease-modifying therapies. Encouraging data from gene therapy trials in patients with Leber hereditary optic neuropathy and advances in DNA editing techniques have raised expectations that successful clinical transition of genetic therapies for PMDs is feasible. However, obstacles to the clinical application of genetic therapies in PMDs remain; the development of innovative, safe and effective genome editing technologies and vectors will be crucial to their future success and clinical approval. In this Perspective, we review progress towards the genetic treatment of nuclear and mitochondrial DNA-related PMDs. We discuss advances in mitochondrial DNA editing technologies alongside the unique challenges to targeting mitochondrial genomes. Last, we consider ongoing trials and regulatory requirements.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Atrofia Óptica Hereditária de Leber
/
Doenças Mitocondriais
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article