Lifeboat Foundation

Although the Human Genome Project announced the completed sequencing of 20,000 human genes more than 20 years ago, scientists are still working to grasp how fully formed beings emerge from basic genetic instructions.

Prime editing, a mightier version of CRISPR/Cas9 technology, has been part of rigorous research and development in recent years. Now, U.S. regulators have greenlit the first-ever clinical trial for this technology.

Massachusetts-based Prime Medicine received the go-ahead from the U.S. Food and Drug Administration (FDA) after preclinical data showed that its candidate was able to correct mutations in chronic granulomatous disease (CGD).

CGD is a rare condition and affects around one in 200,000 people worldwide. It is caused by mutations in any of the six genes that code for the molecule nicotinamide adenine dinucleotide phosphate (NADPH), which is responsible for carrying electrons within cells. White blood cells called phagocytes don’t function properly, and as a result, they fail to protect the body from bacterial and fungal infections.

The ability to genetically modify haematopoietic stem cells would allow the durable treatment of a diverse range of genetic disorders but gene delivery to the bone marrow has not been achieved. Here lipid nanoparticles that target and deliver mRNA to 14 unique cells within the bone marrow are presented.

The human genome consists of around 3 billion base pairs and humans are all 99.6% identical in their genetic makeup. That small 0.4% accounts for any difference between one person and another. Specific combinations of mutations in those base pairs hold important clues about the causes of complex health issues, including heart disease and neurodegenerative diseases like schizophrenia.

Two CRISPR tools for combinatorial genetic perturbations reveal gene regulatory networks.

A large team of biomedical researchers affiliated with a host of institutions across the U.S., the U.K., Saudi Arabia and France, has learned more about many of the factors involved in xenotransplantation as they conducted a large number of tests on two brain-dead human patients that had received genetically engineered pig hearts.

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Tuberous sclerosis is a rare genetic disease that causes benign tumors to grow in the brain and other organs. The disease can be mild, or it can cause severe disabilities. Tuberous sclerosis has no cure, but treatments can help symptoms. More info here.

Tuberous sclerosis (TSC) is a rare genetic disease. It causes benign tumors in the brain and other organs. Learn about symptoms and what can help.

A trailblazer in the field of therapeutic genome editing, Fyodor D. Urnov’s research focuses on developing medicines for devastating genetic diseases.