Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases.

ABSTRACT: Rare diseases pose a global challenge, in that their collective impact on health systems is considerable, whereas their individually rare occurrence impedes research and development of efficient therapies. In consequence, patients and their families are often unable to find an expert for their affliction, let alone a cure. The tide is turning as pharmaceutical companies embrace gene therapy development and as serviceable tools for the repair of primary mutations separate the ability to create cures from underlying disease expertise. Whereas gene therapy by gene addition took decades to reach the clinic by incremental disease-specific refinements of vectors and methods, gene therapy by genome editing in its basic form merely requires certainty about the causative mutation. Suddenly we move

SUBMITTER: Papasavva P 

PROVIDER: S-EPMC6469594 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature