Epidermolysis bullosa (EB) is an inherited, heterogeneous group of rare genetic dermatoses
characterized by mucocutaneous fragility and blister formation, inducible by often minimal …

Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

Treatment of cancer with allogeneic natural killer (NK) cell therapies has seen rapid
development, especially use against hematologic malignancies. Clinical trials of NK cell …

Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that
will drive biological discovery and transform the field of cell and gene therapy. Here, we …

The field of gene therapy has made significant strides over the last several decades toward
the treatment of previously untreatable genetic disease. Gene therapy techniques have …

Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system
represent two instruments of basic and translational research, which both allow to acquire …

The promise of human induced pluripotent stem cells (iPSCs) lies in their ability to serve as
a starting material for autologous, or patient‐specific, stem cell–based therapies. Since the …

Recessive dystrophic epidermolysis bullosa (RDEB) is a severe skin fragility disorder
caused by loss-of-function mutations in the COL7A1 gene, which encodes type VII collagen …

A single gene mutation can cause a number of human diseases that affect the quality of life.
Until the development of clustered regularly interspaced short palindromic repeats …

Base editing introduces precise single-nucleotide edits in genomic DNA and has the
potential to treat genetic diseases such as the blistering skin disease recessive dystrophic …