Gregory Davis, PhD, Vice President of Genome Engineering at Sangamo Therapeutics, explains why the use of genomic medicines, such as Sangamo’s proprietary zinc finger protein technology, are complementary to many rare diseases.
Zinc finger proteins are naturally occurring human DNA-binding proteins and may be engineered to specifically bind any genomic sequence. When fused to transcriptional regulatory domains to form zinc finger protein transcription factors, they can be used to modulate the expression level of the targeted gene. This gives this technology the potential to target a variety of diseases caused by single-gene diseases.
As Dr. Davis explains, many rare diseases are caused by mutations in specific genomic sequences. As such, Sangamo’s zinc finger platform has the potential to be highly suitable for treating these diseases.
To stay abreast with the latest news in rare disease, sign up for one of our newsletters.