[HTML][HTML] Hematopoietic stem cell gene therapy: progress and lessons learned
RA Morgan, D Gray, A Lomova, DB Kohn - Cell stem cell, 2017 - cell.com
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …
has become a clinical standard but is limited by the availability of suitable matched donors …
Gene editing of human hematopoietic stem and progenitor cells: promise and potential hurdles
Hematopoietic stem and progenitor cells (HSPCs) have great therapeutic potential because
of their ability to both self-renew and differentiate. It has been proposed that, given their …
of their ability to both self-renew and differentiate. It has been proposed that, given their …
[HTML][HTML] A comparison of DNA repair pathways to achieve a site-specific gene modification of the Bruton's tyrosine kinase gene
DH Gray, J Santos, AG Keir, I Villegas… - … Therapy-Nucleic Acids, 2022 - cell.com
Gene editing utilizing homology-directed repair has advanced significantly for many
monogenic diseases of the hematopoietic system in recent years but has also been …
monogenic diseases of the hematopoietic system in recent years but has also been …
[图书][B] Gene Editing of Bruton's Tyrosine Kinase for Treatment of X-Linked Agammaglobulinemia
D Gray - 2020 - search.proquest.com
Abstract X-Linked Agammaglobulinemia (XLA) is a primary immunodeficiency characterized
by a lack of mature B lymphocytes and antibody production. Patients with XLA have loss of …
by a lack of mature B lymphocytes and antibody production. Patients with XLA have loss of …