Περίληψη:
beta-thalassemias constitute hereditary blood disorders characterized by
reduced or absence of beta-globin synthesis resulting in mild to severe
anemia, depending on the genotype. More than 200 mutations in the
beta-globin gene are responsible for their specific features leading to
a very heterogeneous phenotype. Current therapies for beta-thalassemia
include blood transfusions, usually along with iron chelation and in
selected cases with bone marrow transplantation (BMT) of HLA-matched
hematopoietic stem cells (HSCs). However, these approaches are limited
by factors, such as iron overload and donor availability, respectively.
Since 2000, when globin lentiviral vectors (LVs) were first successfully
tested for transfer efficiency of the therapeutic transgene, which led
to disease amelioration in murine models, attention was drawn towards
the improvement of such vectors for beta-thalassemia gene therapy.
Constantly improving vector design and efficient HSC manipulation led
recently to the first successful clinical trial in France, which further
proved that this genetic approach can be curative. Furthermore, improved
new efficient vectors and methods to safely monitor integration sites
and therapeutic transgene position effects, promise a new era for
beta-thalassemia gene therapy, with more and safer clinical trials yet
to come.
Συγγραφείς:
Drakopoulou, E.
Papanikolaou, E.
Georgomanoli, M.
Anagnou,
N. P.
