Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency.
J Inherit Metab Dis
; 47(3): 476-493, 2024 05.
Article
en En
| MEDLINE
| ID: mdl-38581234
ABSTRACT
Neurodevelopment is a highly organized and complex process involving lasting and often irreversible changes in the central nervous system. Inherited disorders of neurotransmission (IDNT) are a group of genetic disorders where neurotransmission is primarily affected, resulting in abnormal brain development from early life, manifest as neurodevelopmental disorders and other chronic conditions. In principle, IDNT (particularly those of monogenic causes) are amenable to gene replacement therapy via precise genetic correction. However, practical challenges for gene replacement therapy remain major hurdles for its translation from bench to bedside. We discuss key considerations for the development of gene replacement therapies for IDNT. As an example, we describe our ongoing work on gene replacement therapy for succinic semialdehyde dehydrogenase deficiency, a GABA catabolic disorder.
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Transmisión Sináptica
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Succionato-Semialdehído Deshidrogenasa
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Errores Innatos del Metabolismo de los Aminoácidos
Límite:
Animals
/
Humans
Idioma:
En
Revista:
J Inherit Metab Dis
Año:
2024
Tipo del documento:
Article
País de afiliación:
Estados Unidos