Natural gene therapy in hematopoietic disorders: GATA too

Onderzoeksoutput: Bijdrage aan tijdschriftArtikel

2 Citaten (Scopus)


In this issue of Blood, Catto et al1 describe for the first time the occurrence of somatic genetic rescue (SGR) in an asymptomatic individual carrying a pathogenic germline mutation in GATA2. The authors hypothesize that early recovery of hematopoiesis may also have prevented extrahematological features of GATA2 deficiency in this individual. The finding of SGR in GATA2 deficiency stresses once more that awareness for this phenomenon is needed in genetic counseling and testing of pre- or asymptomatic individuals in families with hereditary bone marrow failure.

Originele taal-2Engels
Pagina's (van-tot)923-924
Aantal pagina's2
Nummer van het tijdschrift8
StatusGepubliceerd - 20 aug. 2020


Duik in de onderzoeksthema's van 'Natural gene therapy in hematopoietic disorders: GATA too'. Samen vormen ze een unieke vingerafdruk.

Citeer dit