gene

Plugging the Gaps in the Human Genome

Supercomputing Helps IRP Researchers Complete Our Genetic Blueprints

DNA sequence

While the Human Genome Project accomplished a remarkable feat in sequencing all the genes in the human genome, technological limitations still left significant swaths of our genetic blueprints unexplored. Recent advances in DNA sequencing are starting to fill in those gaps, but these new technologies require new computational tools to make sense of the data they generate. That’s where computer scientists like the IRP’s Adam Phillippy, Ph.D., come in.

Cutting-Edge Technique Simultaneously Edits Multiple Genetic Targets

Alternative to CRISPR/Cas9 May Cause Fewer Undesired Changes

diagram of DNA strand

IRP researchers have always worked on the cutting edge of biomedical science, from testing the first successful treatment for childhood schizophrenia to pioneering the first screening technique for HIV. In a new study, an IRP team recently achieved yet another first: simultaneously editing two genetic sites in mice using a brand-new approach called base editing that may prove to be more precise – and therefore safer – than other gene editing methods.

Lab-Designed Virus Shows Promise for Inner Ear Gene Therapy

Delivery Method Could Eventually Help Correct Mutations That Cause Hearing Loss

inner ear cells infected by a lab-designed virus with a gene that produces a glowing green protein

Most people probably think of viruses as villains that bring illnesses like measles, HIV, and the flu, but some viruses are proving to be valuable allies in the fight against genetic diseases. In a new study, a team of scientists from the NIH IRP and their colleagues showed the promise of a lab-designed virus for delivering gene therapies aimed at correcting hereditary hearing loss.