Gene therapy using haematopoietic stem and progenitor cells
G Ferrari, AJ Thrasher, A Aiuti - Nature Reviews Genetics, 2021 - nature.com
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …
treatment modality for monogenic disorders of the blood system such as primary …
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission
Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …
condition) have premature deaths and preventable severe chronic complications, which …
Potent and uniform fetal hemoglobin induction via base editing
Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …
cell disease. We compared five strategies in CD34+ hematopoietic stem and progenitor …
Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing
Genome editing has therapeutic potential for treating genetic diseases and cancer.
However, the currently most practicable approaches rely on the generation of DNA double …
However, the currently most practicable approaches rely on the generation of DNA double …
[HTML][HTML] Global marine microbial diversity and its potential in bioprospecting
The past two decades has witnessed a remarkable increase in the number of microbial
genomes retrieved from marine systems,. However, it has remained challenging to translate …
genomes retrieved from marine systems,. However, it has remained challenging to translate …
Sickle cell disease: from genetics to curative approaches
G Hardouin, E Magrin, A Corsia… - Annual Review of …, 2023 - annualreviews.org
Sickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …
gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes …
CRISPRthripsis: The risk of CRISPR/Cas9-induced chromothripsis in gene therapy
Abstract The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9
nuclease system has allowed the generation of disease models and the development of …
nuclease system has allowed the generation of disease models and the development of …
[HTML][HTML] The promise and the hope of gene therapy
E Papanikolaou, A Bosio - Frontiers in genome editing, 2021 - frontiersin.org
It has been over 30 years that visionary scientists came up with the term “Gene Therapy”
suggesting that for certain indications, mostly monogenetic diseases, substitution of the …
suggesting that for certain indications, mostly monogenetic diseases, substitution of the …
Modulating gene regulation to treat genetic disorders
Over a thousand diseases are caused by mutations that alter gene expression levels. The
potential of nuclease-deficient zinc fingers, TALEs or CRISPR fusion systems to treat these …
potential of nuclease-deficient zinc fingers, TALEs or CRISPR fusion systems to treat these …
[HTML][HTML] The power and the promise of CRISPR/Cas9 genome editing for clinical application with gene therapy
N Guo, JB Liu, W Li, YS Ma, D Fu - Journal of Advanced Research, 2022 - Elsevier
Abstract Background Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)
is derived from the bacterial innate immune system and engineered as a robust gene-editing …
is derived from the bacterial innate immune system and engineered as a robust gene-editing …