What is Gene Editing?
Gene editing or genome (a gene consists of DNA to code one protein whereas a ‘genome’ refers to entire sum of DNA in an organism) editing has attracted the attention of the scientific community for its potential to treat human disease. CRISPR (pronounced “crisper”) is one of the most promising gene editing technologies and is gaining widespread appeal from many researchers.
Why is everyone talking about CRISPR?
CRISPR, or Clustered Regularly Interspaced Short Palindromic Repeats, are repeating DNA sequences originally discovered in bacteria in 1987. Bacteria use these sequences along with the enzyme Cas9 (enzymes are produced by organisms to act as a ‘spark’ to create certain biochemical reactions) to protect them against future viruses. Scientists have since used the CRISPR-Cas9 system to recognize and cut targeted DNA sequences in human cells as well as other organisms, enabling the study of specific genes in human biology and disease. CRISPR has the potential to delete diseased genes and insert new, normal genes in their place, impacting the future of cellular therapy.
How will CRISPR be used to treat disease?
Other gene editing technologies such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) have also shown promise, with two clinical trials underway for a ZFN-based technology to treat Hunter’s syndrome, a genetic neurological disease [ref]. Although CRISPR still requires further development and safety evaluation to assess off-target effects, it has emerged as the leading gene editing technology for many researchers with over 14,000 scientific publications citing the use of CRISPR in 2017 [ref]. The potential of CRISPR for the precise treatment of genetic disorders including sickle-cell anemia, cystic fibrosis, and Duchenne’s muscular dystrophy has revolutionized the field of genome editing.
What can I do to contribute?
Gene editing often starts with reprogramming stem cells, which are rare compared to most cell types and found most frequently in cord blood and bone marrow. Donations of bone marrow provide healthy control samples for researchers to perform CRISPR experiments, instead of relying on sources such as peripheral blood where the frequency of these rare cells is very low. Access to bone marrow specimens means that researchers can spend less time trying to find the right samples and focus instead on discovering the next breakthrough gene editing technology.
