Add to ORKGThe CRISPR/Cas9 DNA editing system offers the potential for revolutionary new treatments of genetic diseases. Pioneering work by Carroll set the stage for this revolution by characterizing how cells detect and repair double-stranded DNA breaks. Sickle cell disease is common among African Americans and very widespread in tropical regions of Africa and southern Asia. Carroll and his colleagues used the CRISPR/Cas9 gene editing system and a novel delivery method to correct the sickle mutation in adult human hematopoietic stem/progenitor cells. Continuing work on this approach has improved the efficiency of correction, and treatment of the first patients is anticipated by the end of 2020
The CRISPR/Cas9 prokaryotic adaptive immune system and its swift repurposing for genome editing enab...
BackgroundA point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subuni...
Kmiec, EricSingle base mutations can be repaired by introducing single stranded DNA oligonucleotides...
The CRISPR/Cas9 DNA editing system offers the potential for revolutionary new treatments of genetic ...
Background: Sickle cell disease is a genetic disorder that causes several morbidities and increased ...
Genetic diseases of blood cells are prime candidates for treatment through ex vivo gene editing of C...
Site-specific correction of a point mutation causing a monogenic disease in autologous hematopoietic...
According to current research, CRISPR/Cas9-mediated genome editing has shown enormous potential in t...
Abstract Background Sickle cell disease (SCD) is a disorder of red blood cells (RBCs) expressing abn...
Sickle cell disease (SCD) is an autosomal recessive disorder caused by aberrant hemoglobin structure...
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated system (Cas9)-m...
Sickle cell disease (SCD) is an inherited red blood cell disorder that affects approximately 100,000...
Background A point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subu...
Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach for ge...
The CRISPR/Cas9 prokaryotic adaptive immune system and its swift repurposing for genome editing enab...
The CRISPR/Cas9 prokaryotic adaptive immune system and its swift repurposing for genome editing enab...
BackgroundA point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subuni...
Kmiec, EricSingle base mutations can be repaired by introducing single stranded DNA oligonucleotides...
The CRISPR/Cas9 DNA editing system offers the potential for revolutionary new treatments of genetic ...
Background: Sickle cell disease is a genetic disorder that causes several morbidities and increased ...
Genetic diseases of blood cells are prime candidates for treatment through ex vivo gene editing of C...
Site-specific correction of a point mutation causing a monogenic disease in autologous hematopoietic...
According to current research, CRISPR/Cas9-mediated genome editing has shown enormous potential in t...
Abstract Background Sickle cell disease (SCD) is a disorder of red blood cells (RBCs) expressing abn...
Sickle cell disease (SCD) is an autosomal recessive disorder caused by aberrant hemoglobin structure...
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated system (Cas9)-m...
Sickle cell disease (SCD) is an inherited red blood cell disorder that affects approximately 100,000...
Background A point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subu...
Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach for ge...
The CRISPR/Cas9 prokaryotic adaptive immune system and its swift repurposing for genome editing enab...
The CRISPR/Cas9 prokaryotic adaptive immune system and its swift repurposing for genome editing enab...
BackgroundA point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subuni...
Kmiec, EricSingle base mutations can be repaired by introducing single stranded DNA oligonucleotides...