Title:New Frontiers in Cystic Fibrosis Therapy: The Case of Stem Cells
Volume: 3
Issue: 2
Author(s): Guoshun Wang*
Affiliation:
- Department of Microbiology, Immunology and Parasitology, Department of Genetics and Department of Medicine, Louisiana State University Health Sciences Center CSRB 642, 533 Bolivar Street, New Orleans, LA 70112,United States
Keywords:
Cystic fibrosis, gene therapy, stem cells, CRISPR/Cas9 genomic editing, CF transmembrane conductance regulator
gene (CFTR), lung pathology.
Abstract: Cystic fibrosis (CF) is an autosomal recessive inherited disorder that affects ~80,000 individuals
worldwide. Mutations in the CF transmembrane conductance regulator gene (CFTR) cause dysfunction
of the CFTR protein, a cAMP- and PKA-activated chloride channel. Decades of research and
development have made great progress in molecular understanding and clinical care of this disease,
which has dramatically extended the patient median life expectancy to over 40 years. However, this
devastating disease remains incurable, with lung pathology the most common cause of morbidity and
mortality. Stem cells maintain the capacity of self-renewal and induced differentiation, thus holding
promise for CF therapy. While early studies generated the excitement of lung engraftment and regeneration
by different origins of stem cells, several encountered obstacles impede viable development of this
strategy into a feasible therapy. This review article will provide an update of the research and discuss
the major challenges facing the field. Moreover, new emerging technologies, such as CRISPR/Cas9 genomic
editing, make possible the precise correction of the CFTR defect in situ. Potential impacts of
these advancements on future prospects of CF stem cell therapy will be highlighted.
