The World Health Organization reports that around 13 percent of the world’s population is classified as “obese.” Today, medical experts know that obesity is much more than just a battle with overeating. In fact, obesity is a medical condition largely affected by an individuals’ genetics. As researchers work to develop drugs to treat obesity, exploring …
Chimpanzee DNA Sheds Light on Human Brain Evolution
In evolutionary terms, chimpanzees are humans’ closest living relatives. But how close are we, really? Science Daily reports that researchers at Lund University in Sweden used stem cells to explore the differences between human brains and chimp brains. During the study, the researchers managed to identify a previously overlooked part of our DNA. That study, …
Can Gene Therapy Cure Sickle Cell Disease?
Despite being one of the world’s most common inherited blood disorders, sickle cell disease has few treatment options, even with the approval of three new drugs since 2017. Several companies hope to use new gene-editing tools to cure the disease by targeting the mutations that cause it. One such treatment is in the final stage …
New Hope for Ultra-Rare Childhood Disease
The recent release of findings from a small Phase 1 clinical trial not only gives hope to families affected by an ultra-rare childhood disease, but may also offer a glimpse into future treatment of genetic disorders affecting the central nervous system. If the study continues to yield results as researchers have seen, these findings may …
CRISPR and the Continuing Quest for CAR-T Safety and Durability
CAR-T is an immunotherapy that is currently used to treat blood cancers and is in clinical trials to treat other cancers as well. This treatment requires harvesting a patient’s T cells from their blood, genetically modifying them to be more efficient in attacking cancer cells, growing them in the lab and then infusing them back …
mRNA: Great Expectations vs. Limited Applications
While research on potential therapeutic applications of mRNA has been underway for more than a decade, the success of the Pfizer and Moderna COVID-19 vaccines has expanded interest in the field, with manufacturers like Sanofi announcing significant investment and intent to become an active participant in developing mRNA-based therapies. In addition to developing antibodies as …
Move Over AAV – Anelloviruses May Be Key to NextGen Gene Therapy
Biotech start-up Ring Therapeutics, founded by Flagship Pioneering in 2017, hopes to transform gene therapy by using a new class of vector — anelloviruses – a diverse family of harmless viruses present in the human body. Current gene therapies mostly use an adeno-associated virus (AAV) to carry genetic material into a cell – often a …
A Genetic Game-Changer in Treating Obesity
An estimated 650 million adults live with obesity today, which puts those individuals at increased risk for some of the leading causes of death worldwide, including diabetes, heart disease, stroke, and some types of cancer. Most prescription weight-loss drugs work by decreasing appetite or providing a feeling of satiety to help people lose weight. The …
Clinical Trial Shows In Vivo CRISPR-Cas9 Therapy Effective Against Rare Disease
Transthyretin amyloidosis (ATTR amyloidosis) is a rare but fatal disease marked by clumping and accumulation of mutated, misfolded transthyretin proteins in peripheral nerves and cardiac tissue. Most patients experience neuropathy and/or cardiomyopathy, and average survival from disease onset is between two and 17 years. About 50,000 people worldwide have its autosomal dominant inherited form. Current …
Could Giraffes Advance Human Genetic Research?
Depending on your perspective, giraffes are either the world’s most graceful animal or a baffling, doe-eyed sloppy eater. (This could depend largely on whether you’re observing giraffes on safari or, say, feeding them pellets at the zoo.) But personal giraffe encounters aside, one thing is certain: these fascinating animals represent several truly astounding evolutionary feats. …
