Τίτλος:
Success Stories and Challenges Ahead in Hematopoietic Stem Cell Gene
Therapy: Hemoglobinopathies as Disease Models
Γλώσσες Τεκμηρίου:
Αγγλικά
Περίληψη:
Gene therapy is a relatively novel field that amounts to around four
decades of continuous growth with its good and bad moments. Currently,
the field has entered the clinical arena with the ambition to fulfil its
promises for a permanent fix of incurable genetic disorders.
Hemoglobinopathies as target diseases and hematopoietic stem cells
(HSCs) as target cells of genetic interventions had a major share in the
research effort toward efficiently implementing gene therapy. Dissection
of HSC biology and improvements in gene transfer and gene expression
technologies evolved in an almost synchronous manner to a point where
the two fields seem to be functionally intercalated. In this review, we
focus specifically on the development of gene therapy for hemoglobin
disorders and look at both gene addition and gene correction strategies
that may dominate the field of HSC-directed gene therapy in the near
future and transform the therapeutic landscape for genetic diseases.
Συγγραφείς:
Yannaki, Evangelia
Psatha, Nikoletta
Papadopoulou, Anastasia and
Athanasopoulos, Takis
Gravanis, Achilleas
Roubelakis, Maria G.
and Tsirigotis, Panagiotis
Anagnostopoulos, Achilles
Anagnou,
Nicholas P.
Vassilopoulos, George
Περιοδικό:
Human Gene Therapy
Εκδότης:
Mary Ann Liebert, Inc. 140 Huguenot Street, 3rd Floor New Rochelle, NY 10801 USA
Λέξεις-κλειδιά:
gene therapy; gene editing; hemoglobinopathies; hematopoietic stem cell;
gene addition; thalassemia
DOI:
10.1089/hum.2021.196
