As a hereditary trait, eye color tends to be an introductory lesson to human genetics. Think back to high school biology class, which often covers the unique genetic circumstances that create eye color. Those lessons typically involve one traditional principle: that the allele that codes for brown is dominant over green or blue. But a …
Understanding How Cells Tell RNA and DNA Apart Could Lead to New Antivirals
RNA viruses are known to cause a large number of diseases in humans, including COVID-19, measles, influenza, hepatitis, yellow fever and Zika. Most new and reemerging viral diseases, in fact, are based on RNA. While vaccines often have been effective against RNA viruses, treatments for disease caused by these viruses can be more challenging. One …
Five Criteria to Use When Selecting a CRO for a Gene Therapy Study
Contract research organizations (CROs) have been an integral part of drug development since the 1920s, first as providers of preclinical testing services, then as organizations that could manage clinical trials on behalf of their sponsors. While this makes the majority of CROs knowledgeable experts in the management of clinical trials, especially for traditional small-molecule therapies, …
How a Rare Breed of Rabbit Is Advancing Genetics
Although you might think of the ability to hop as one of rabbits’ defining characteristics, some rabbits don’t hop at all. The sauteur d’Alfort is a rare breed of domestic rabbit that sometimes walks on its front paws with its hind legs in the air. Its inability to hop and aptitude for handstands has fascinated scientists for years. …
Determining “Responsible Clinical Use” in Heritable Human Genome Editing
A common refrain in science fiction movies is the cautionary statement, “Just because we can doesn’t mean we should.” As new technologies such as CRISPR/Cas9 have made gene editing at precise locations in DNA possible, that sentiment has plagued ethicists and scientists alike regarding heritable human genomes. The need for guidelines became apparent when Dr. …
Robust Cell and Gene Therapy Pipeline Targets a Range of Diseases
Cell and gene therapies (CGTs) are quickly making their way from theory to clinical practice, primarily because they target specific disease drivers rather than the broad targets of small-molecule therapies. This specificity is possible because cell and gene therapies target genes and cells to change the way tissues function. The goal for both types of …
The Genetics of Calico Cats
Whether you adore cats or you’re allergic to them, you might be surprised to learn that you can better understand genetics by studying calico cats. More specifically, you can learn about epigenetics, or the study of changes in organisms due to the modification of gene expression, not the alteration of DNA. Known for their distinctive patches …
Could Gene Therapy Hold the Answer to Danon Disease?
Danon Disease is a rare, life-threatening condition in which the body’s protein removal and recycling processes no longer work properly. The result is dysfunction of the heart, skeletal muscle, nervous system, eyes, and liver. The disease is more common in males and is thought to be inherited. Symptoms begin in early childhood or adolescence, and …
Gene Therapy and Inherited Retinal Diseases
Inherited retinal diseases (IRDs) can cause vision loss that ranges from mild to severe, and can even cause blindness. These diseases affect people of all ages, and different IRDs progress at different rates. They are degenerative and get worse over time. Common IRDs include: Leber congenital amaurosis (LCA) Retinitis pigmentosa Choroideremia Stargardt disease Achromatopsia IRDs …
Squid Genes: Self-Edits in the Wild and CRISPR Edits in the Lab
Squid have long been a key organism for deepening the understanding of how nerves transmit signals. They have been passed over for gene-editing studies, however, as their genes were thought to be intractable. But a group of researchers led by Joshua Rosenthal, a biologist at the Marine Biological Laboratory in Woods Hole, Massachusetts, has found …
