A major obstacle to CRISPR/Cas9 – preexisting immunity

The discovery of CRISPR/Cas9 is one of the biggest scientific breakthroughs in recent memory. This technology, first discovered in microbes, enables the cleaving of DNA in order to remove or replace existing genes. For a crash course into the history of CRISPR/Cas9 discovery and development with primary sources please refer to the footnote*. This technology will enable us cure many genetic diseases. Work is currently … Continue reading A major obstacle to CRISPR/Cas9 – preexisting immunity

Viruses, not all are bad for you

When you think of viruses, the yearly flu or even the Ebola or Swine flu outbreaks may come to mind. However, not all viruses cause disease – some even provide cures! Adeno-associated virus (AAV) can infect humans, but is not known to cause disease. In other words, this virus is good at getting its genetic information (genes) into human cells. What if its genes were … Continue reading Viruses, not all are bad for you

CRISPR 2.0: Genome engineering made easy as A-B-C

CRISPR 2.0 is causing quite the ruckus in the scientific community. Why? Imagine that you had written a note in permanent marker, but later decided you wanted to change a single word. Without the ability to erase, your options would be limited, and further changes might make the note illegible. New CRISPR technologies, or “base editors,” behave as molecular erasers. These molecular erasers enable you to very precisely … Continue reading CRISPR 2.0: Genome engineering made easy as A-B-C

Masquerade

Gene therapy is an approach to treating genetic diseases by re-introducing a functional copy of a gene into cells to replace the mutated, disease-causing gene. To get these genes into the cells, scientists create a vector by packaging the DNA encoding the correct gene into the outer shell–called the capsid–of a virus. A vector retains the ability of the virus to enter human cells and … Continue reading Masquerade

Mouse embryo

Gene editing emerges as a new therapeutic strategy for Duchenne muscular dystrophy

Advances in gene editing technology have spurred considerable progress towards a treatment for Duchenne muscular dystrophy (DMD).  Although the disease is rare – affecting roughly 1 in 5,000 male births – its consequences are devastating: patients are confined to wheelchairs at an early age and often succumb to heart or respiratory failure in their twenties or thirties.  No treatments are currently available, but three separate … Continue reading Gene editing emerges as a new therapeutic strategy for Duchenne muscular dystrophy

Why ‘3-parent babies?’ DNA from Mom and Dad. Healthy mitochondria from another.

Mitochondria provide the energy for cells, but unlike DNA, they are only inherited from our mothers through their eggs. Defective mitochondria cause diseases which are incurable in adulthood. However, they are treatable before conception with “cytoplasmic transfer,” where defective mitochondria are swapped out for healthy ones. Continue reading Why ‘3-parent babies?’ DNA from Mom and Dad. Healthy mitochondria from another.