Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is
now commonplace in academic laboratories and industry for both research and clinical …

The frontiers of precision medicine have been revolutionized by the development of
Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR)/Cas9 as an editing …

More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …

CRISPR/Cas technology has revolutionized the fields of the genome-and epigenome-
editing by supplying unparalleled control over genomic sequences and expression …

Achieving the full potential of zinc-finger nucleases (ZFNs) for genome engineering in
human cells requires their efficient delivery to the relevant cell types. Here we exploited the …

The concept of gene therapy originated in the mid twentieth century and was perceived as a
revolutionary technology with the promise to cure almost any disease of which the molecular …

Lentiviral vectors (LVs) have emerged as potent and versatile vectors for ex vivo or in vivo
gene transfer into dividing and nondividing cells. Robust phenotypic correction of diseases …

The abilities of stem cells to self-renew and form different mature cells expand the
possibilities of applications in cell-based therapies such as tissue recomposition in …

The capacity to efficiently transduce nondividing cells, shuttle large genetic payloads, and
maintain stable long-term transgene expression are attributes that have brought lentiviral …

The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …