[Gene Therapies in Motor Neuron Diseases ALS and SMA]. / Gentherapien bei den Motoneuronerkrankungen ALS und SMA.
Fortschr Neurol Psychiatr
; 91(4): 153-163, 2023 Apr.
Article
em De
| MEDLINE
| ID: mdl-36822211
ABSTRACT
In the past, the diagnosis of motor neuron diseases such as amyotrophic lateral sclerosis (ALS) and 5q-associated spinal muscular atrophy (SMA) meant powerlessness in the face of seemingly untreatable diseases with severe motor-functional limitations and sometimes fatal courses. Recent advances in an understanding of the genetic causalities of these diseases, combined with success in the development of targeted gene therapy strategies, spell hope for effective, innovative therapeutic approaches, pioneering the ability to treat neurodegenerative diseases. While gene therapies have been approved for SMA since a few years, gene therapy research in ALS is still in clinical trials with encouraging results. This article provides an overview of the genetic background of ALS and SMA known to date and gene therapy approaches to them with a focus on therapy candidates that are in clinical trials or have already gained market approval.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Atrofia Muscular Espinal
/
Doença dos Neurônios Motores
/
Esclerose Lateral Amiotrófica
Tipo de estudo:
Diagnostic_studies
Limite:
Humans
Idioma:
De
Revista:
Fortschr Neurol Psychiatr
Ano de publicação:
2023
Tipo de documento:
Article