A series of recent discoveries harnessing the adaptive immune system of prokaryotes to
perform targeted genome editing is having a transformative influence across the biological …

Recent progress in developing programmable nucleases, such as zinc-finger nucleases,
transcription activator–like effector nucleases (TALENs) and clustered regularly interspaced …

This will be a substantial revision of a good selling text for upper division/first graduate
courses in biomedical transport phenomena, offered in many departments of biomedical and …

Editing the β-globin locus in hematopoietic stem cells is an alternative therapeutic approach
for gene therapy of β-thalassemia and sickle cell disease. Using the CRISPR/Cas9 system …

Molecular medicine has entered a high-tech age that provides curative treatments of
complex genetic diseases through genetically engineered cellular medicinal products. Their …

Beta-thalassemia is one of the most common recessive genetic diseases, caused by
mutations in the HBB gene. Over 200 different types of mutations in the HBB gene …

Background Natural killer (NK) cells hold great promise in treating diverse hematopoietic
and solid tumors. Despite their availability from peripheral blood and cord blood, stem cell …

Genome engineering technologies right from viral vector-mediated to protein-based editing—
which include zinc finger nucleases, TALENs, and CRISPR/Cas systems—have been …

Clustered regularly interspaced short palindromic repeats (CRISPR)-caspase 9 (Cas9)
genome editing technology holds great promise for the field of human gene therapy …

Hematopoietic stem cells (HSCs) have been used for therapeutic purposes for decades in
the form of autologous and allogeneic transplantation and are currently emerging as an …