Genome editing based on sequence-specific designer nucleases, also known as
programmable nucleases, seeks to modify in a targeted and precise manner the genetic …

Genome editing refers to a process by which a specific chromosomal sequence is changed.
The edited chromosomal sequence may comprise an insertion of at least one nucleotide, a …

Gene therapy currently in development for hemoglobinopathies utilizes ex vivo lentiviral
transduction of CD34+ hematopoietic stem and progenitor cells (HSPCs). A small-molecule …

Despite the rapid development of CRISPR/Cas9-mediated gene editing technology, the
gene editing potential of CRISPR/Cas9 is hampered by low efficiency, especially for clinical …

Background Given known inefficiencies in reprogramming of fibroblasts into mature induced
cardiomyocytes (iCM s), we sought to identify small molecules that would overcome these …

Integrase-defective lentiviral vectors (IDLVs) have been used as a safe and efficient delivery
system in several immunization protocols in murine and non-human primate preclinical …

Chromosomal structure of nuclear DNA is usually maintained by insertion of nucleosomes
into preexisting chromatin, both on newly synthesized DNA at replication forks and at sites of …

Genome-edited human induced pluripotent stem cells (iPSCs) hold great promise for
therapeutic applications. However, low editing efficiency has hampered the applications of …

Gene editing enables the site-specific modification of the genome. These technologies have
rapidly advanced such that they have entered common use in experimental hematology to …

Human bone marrow‐derived mesenchymal stromal cells (MSCs) obtained from aged
patients are prone to senesce and diminish their differentiation potential, therefore limiting …