Towards gene therapy for IPEX syndrome.
Eur J Immunol
; 52(5): 705-716, 2022 05.
Article
em En
| MEDLINE
| ID: mdl-35355253
ABSTRACT
Immune dysregulation polyendocrinopathy enteropathy X linked (IPEX) syndrome is an uncurable disease of the immune system, with immune dysregulation that is caused by mutations in FOXP3. Current treatment options, such as pharmacological immune suppression and allogeneic hematopoietic stem cell transplantation, have been beneficial but present limitations, and their life-long consequences are ill-defined. Other similar blood monogenic diseases have been successfully treated using gene transfer in autologous patient cells, thus providing an effective and less invasive therapeutic. Development of gene therapy for patients with IPEX is particularly challenging because successful strategies must restore the complex expression profile of the transcription factor FOXP3, ensuring it is tightly regulated and its cell subset-specific roles are maintained. This review summarizes current efforts toward achieving gene therapy to treat immune dysregulation in IPEX patients.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Doenças Genéticas Ligadas ao Cromossomo X
/
Diabetes Mellitus Tipo 1
/
Doenças do Sistema Imunitário
Limite:
Humans
Idioma:
En
Revista:
Eur J Immunol
Ano de publicação:
2022
Tipo de documento:
Article
País de afiliação:
Estados Unidos