Highly efficient therapeutic gene editing of human hematopoietic stem cells
Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …
ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by …
Targeted gene delivery: where to land
G Pavani, M Amendola - Frontiers in genome editing, 2021 - frontiersin.org
Genome-editing technologies have the potential to correct most genetic defects involved in
blood disorders. In contrast to mutation-specific editing, targeted gene insertion can correct …
blood disorders. In contrast to mutation-specific editing, targeted gene insertion can correct …
[HTML][HTML] Recent advances in genome editing of stem cells for drug discovery and therapeutic application
Genome engineering technologies right from viral vector-mediated to protein-based editing—
which include zinc finger nucleases, TALENs, and CRISPR/Cas systems—have been …
which include zinc finger nucleases, TALENs, and CRISPR/Cas systems—have been …
Removal of TREX1 activity enhances CRISPR–Cas9-mediated homologous recombination
Abstract CRISPR–Cas9-mediated homology-directed repair (HDR) can introduce desired
mutations at targeted genomic sites, but achieving high efficiencies is a major hurdle in …
mutations at targeted genomic sites, but achieving high efficiencies is a major hurdle in …
Adenine base editing efficiently restores the function of Fanconi anemia hematopoietic stem and progenitor cells
SM Siegner, L Ugalde, A Clemens… - Nature …, 2022 - nature.com
Fanconi Anemia (FA) is a debilitating genetic disorder with a wide range of severe
symptoms including bone marrow failure and predisposition to cancer. CRISPR-Cas …
symptoms including bone marrow failure and predisposition to cancer. CRISPR-Cas …
Diversifying the structure of zinc finger nucleases for high-precision genome editing
DE Paschon, S Lussier, T Wangzor, DF Xia… - Nature …, 2019 - nature.com
Genome editing for therapeutic applications often requires cleavage within a narrow
sequence window. Here, to enable such high-precision targeting with zinc-finger nucleases …
sequence window. Here, to enable such high-precision targeting with zinc-finger nucleases …
NHEJ-mediated repair of CRISPR-Cas9-induced DNA breaks efficiently corrects mutations in HSPCs from patients with fanconi anemia
FJ Román-Rodríguez, L Ugalde, L Álvarez, B Díez… - Cell Stem Cell, 2019 - cell.com
Non-homologous end-joining (NHEJ) is the preferred mechanism used by hematopoietic
stem cells (HSCs) to repair double-stranded DNA breaks and is particularly increased in …
stem cells (HSCs) to repair double-stranded DNA breaks and is particularly increased in …
Integrin-α3 is a functional marker of ex vivo expanded human long-term hematopoietic stem cells
E Tomellini, I Fares, B Lehnertz, J Chagraoui… - Cell Reports, 2019 - cell.com
Transplantation of expanded hematopoietic stem cells (HSCs) and gene therapy based on
HSC engineering have emerged as promising approaches for the treatment of …
HSC engineering have emerged as promising approaches for the treatment of …
CRISPR/Cas9-mediated in situ correction of LAMB3 gene in keratinocytes derived from a junctional epidermolysis bullosa patient
D Benati, F Miselli, F Cocchiarella, C Patrizi… - Molecular Therapy, 2018 - cell.com
Deficiency of basement membrane heterotrimeric laminin 332 component, coded by LAMA3,
LAMB3, and LAMC2 genes, causes junctional epidermolysis bullosa (JEB), a severe skin …
LAMB3, and LAMC2 genes, causes junctional epidermolysis bullosa (JEB), a severe skin …
Therapy development by genome editing of hematopoietic stem cells
L Koniali, CW Lederer, M Kleanthous - Cells, 2021 - mdpi.com
Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …
blood and immune systems has placed them at the forefront of cell and gene therapy …