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.
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.
Denis Noble discusses common misconceptions in genetics. Are our genes really as deterministic as we think they are?Watch the full talk] at https://iai.tv/vid…
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I met Prof. Ed Boyden at last year’s Global Future 2045 conference in New York. There I was highly impressed with Boyden’s impressive work in neuroscience in general and optogenetics in particular, as well as the profound implications it would have on our ability to understand and manipulate the brain. And so I knew instantly I must bring him for an interview.
Genomics is revolutionizing medicine and science, but current approaches still struggle to capture the breadth of human genetic diversity. Pangenomes that incorporate many people’s DNA could be the answer, and a new project thinks quantum computers will be a key enabler.
When the Human Genome Project published its first reference genome in 2001, it was based on DNA from just a handful of humans. While less than one percent of our DNA varies from person to person, this can still leave important gaps and limit what we can learn from genomic analyses.
That’s why the concept of a pangenome has become increasingly popular. This refers to a collection of genomic sequences from many different people that have been merged to cover a much greater range of human genetic possibilities.