Translational research for bone marrow failure patients.
Exp Hematol
; 105: 18-21, 2022 01.
Article
en En
| MEDLINE
| ID: mdl-34801643
ABSTRACT
Bone marrow failure syndromes encompass a range of inherited and acquired hematological diseases that result in insufficient blood cell production, which leads to severe complications including anemia, weakening of the immune system, impaired coagulation, and increased risk of cancer. Within inherited bone marrow failure syndromes, a number of genetically distinct diseases have been described including Shwachman-Diamond syndrome and Fanconi anemia. Given the genetic complexity and poor prognosis of these inherited bone marrow failure syndromes, there is increasing interest in both characterizing the genetic landscapes of these diseases and developing novel gene therapies to effectively monitor and cure patients. These topics were the focus of the winter 2021 International Society for Experimental Hematology New Investigator Webinar, which featured presentations by Dr. Akiko Shimamura and Dr. Paula Río. Here, we review the topics covered within this webinar.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Contexto en salud:
1_ASSA2030
Problema de salud:
1_geracao_evidencia_conhecimento
Asunto principal:
Trastornos de Fallo de la Médula Ósea
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Exp Hematol
Año:
2022
Tipo del documento:
Article
País de afiliación:
Reino Unido