RNA drugs and RNA targets for small molecules: principles, progress, and challenges
RNA-based therapies, including RNA molecules as drugs and RNA-targeted small
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …
molecules, offer unique opportunities to expand the range of therapeutic targets. Various …
Next-generation stem cells—ushering in a new era of cell-based therapies
EA Kimbrel, R Lanza - Nature Reviews Drug Discovery, 2020 - nature.com
Naturally occurring stem cells isolated from humans have been used therapeutically for
decades. This has primarily involved the transplantation of primary cells such as …
decades. This has primarily involved the transplantation of primary cells such as …
Human genetic diversity alters off-target outcomes of therapeutic gene editing
CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat
disease. However, standard computational and biochemical methods to predict off-target …
disease. However, standard computational and biochemical methods to predict off-target …
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 …
[HTML][HTML] Deliver the promise: RNAs as a new class of molecular entities for therapy and vaccination
The concepts of develo** RNAs as new molecular entities for therapies have arisen again
and again since the discoveries of antisense RNAs, direct RNA-protein interactions …
and again since the discoveries of antisense RNAs, direct RNA-protein interactions …
[HTML][HTML] Advances in CRISPR/Cas-based gene therapy in human genetic diseases
CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for
introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non …
introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non …
Extinction of all infectious HIV in cell culture by the CRISPR-Cas12a system with only a single crRNA
The CRISPR-Cas9 system has been used for genome editing of various organisms. We
reported inhibition of the human immunodeficiency virus (HIV) in cell culture infections with a …
reported inhibition of the human immunodeficiency virus (HIV) in cell culture infections with a …
Precision editing as a therapeutic approach for β-hemoglobinopathies
Beta-hemoglobinopathies are the most common genetic disorders worldwide, caused by a
wide spectrum of mutations in the β-globin locus, and associated with morbidity and early …
wide spectrum of mutations in the β-globin locus, and associated with morbidity and early …
Adenine base editor–mediated correction of the common and severe IVS1-110 (G> A) β-thalassemia mutation
G Hardouin, P Antoniou, P Martinucci, T Felix… - Blood, 2023 - ashpublications.org
Abstract β-Thalassemia (BT) is one of the most common genetic diseases worldwide and is
caused by mutations affecting β-globin production. The only curative treatment is allogenic …
caused by mutations affecting β-globin production. The only curative treatment is allogenic …
Efficient homology-directed repair with circular single-stranded DNA donors
While genome editing has been revolutionized by the advent of CRISPR-based nucleases,
difficulties in achieving efficient, nuclease-mediated, homology-directed repair (HDR) still …
difficulties in achieving efficient, nuclease-mediated, homology-directed repair (HDR) still …