Therapeutics in paediatric genetic diseases: Current and future landscape
Singapore medical journal
; : 7-16, 2023.
Article
en En
| WPRIM
| ID: wpr-969660
Biblioteca responsable:
WPRO
ABSTRACT
There are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the past 3 decades, there has been rapid advancement in the development of novel therapies, including gene therapy, for many PGDs. The therapeutic success of treatment relies heavily on knowledge of the genetic basis and the disease mechanism. Specifically, gene therapy has been shown to be effective in various clinical trials, and indeed, these trials have led to regulatory approvals, paving the way for gene therapies for other types of PGDs. In this review, we provide an overview of the treatment strategies and focus on some of the recent advancements in therapeutics for PGDs.
Palabras clave
Texto completo:
1
Base de datos:
WPRIM
Asunto principal:
Terapia Genética
/
Enfermedades Genéticas Congénitas
Límite:
Child
/
Humans
Idioma:
En
Revista:
Singapore medical journal
Año:
2023
Tipo del documento:
Article