Advances and challenges in the development of gene therapy medicinal products for rare diseases
JA Bueren, A Auricchio - Human Gene Therapy, 2023 - liebertpub.com
The development of viral vectors and recombinant DNA technology since the 1960s has
enabled gene therapy to become a real therapeutic option for several inherited and …
enabled gene therapy to become a real therapeutic option for several inherited and …
Re-envisioning genetic predisposition to childhood and adolescent cancers
CP Kratz - Nature Reviews Cancer, 2024 - nature.com
Although cancer is rare in children and adolescents, it remains a leading cause of death
within this age range, and genetic predisposition is the main known risk factor. Since the …
within this age range, and genetic predisposition is the main known risk factor. Since the …
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials
Background Allogeneic haematopoietic stem-cell transplantation is the standard treatment
for bone marrow failure (BMF) in patients with Fanconi anaemia, but transplantation …
for bone marrow failure (BMF) in patients with Fanconi anaemia, but transplantation …
Management of Fanconi anemia beyond childhood
TS Olson - Hematology, 2023 - ashpublications.org
Fanconi anemia (FA) has long been considered a severe inherited bone marrow failure
(BMF) disorder of early childhood. Thus, management of this multisystem disorder has …
(BMF) disorder of early childhood. Thus, management of this multisystem disorder has …
Diamond-Blackfan anemia, the archetype of ribosomopathy: How distinct is it from the other constitutional ribosomopathies?
L Da Costa, N Mohandas, L David-NGuyen… - Blood Cells, Molecules …, 2024 - Elsevier
Diamond-Blackfan anemia (DBA) was the first ribosomopathy described in humans. DBA is
a congenital hypoplastic anemia, characterized by macrocytic aregenerative anemia …
a congenital hypoplastic anemia, characterized by macrocytic aregenerative anemia …
[HTML][HTML] Clinical and genetic features of Fanconi anemia associated with a variant of FANCA gene: case report and literature review
L Zhong, W Zhang, K Zhang, C Li, X Mu, Y Chu, Z Gai… - Medicine, 2024 - journals.lww.com
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Why hematopoietic stem cells fail in Fanconi anemia: Mechanisms and models
S Liu, ES Vivona, P Kurre - BioEssays, 2025 - Wiley Online Library
Fanconi anemia (FA) is generally classified as a DNA repair disorder, conferring a genetic
predisposition to cancer and prominent bone marrow failure (BMF) in early childhood …
predisposition to cancer and prominent bone marrow failure (BMF) in early childhood …
[HTML][HTML] Correcting the aberrant Fanconi anemia transcriptional program by gene therapy
Y Dror - Haematologica, 2023 - ncbi.nlm.nih.gov
Fanconi anemia (FA) is a multisystem disorder, but hematopoietic derangements are the
most common causes of morbidity and mortality, which include bone marrow failure and …
most common causes of morbidity and mortality, which include bone marrow failure and …