Lifeboat Foundation

New research suggests that a controversial gene-editing experiment to make children resistant to HIV may also have enhanced their ability to learn and form memories.

Researchers at Tel Aviv University say they have developed a new, noninvasive method of discovering genetic disorders that can let parents find out the health of their fetus as early as 11 weeks into pregnancy.

A simple blood test lets doctors diagnose genetic disorders in fetuses early in pregnancy by sequencing small amounts of DNA in the mother’s and the father’s blood. A computer algorithm developed by the researchers analyzes the results of the sequencing and then produces a “map” of the fetal genome, predicting mutations with 99 percent or better accuracy, depending on the mutation type, the researchers said in a study published Wednesday in Genome Research.

The algorithm is able to distinguish between the genetic material of the parents and that of the fetus, said Prof. Noam Shomron of Tel Aviv University’s Sackler School of Medicine led the research, in a phone interview with The Times of Israel.

Continue reading “Israeli team develops way to find genetic flaws in fetus at 11 weeks” »

Researchers at the Salk Institute have moved a step closer to a possible therapy for Hutchinson-Gilford progeria syndrome, a rare genetic disorder that is often described as accelerated aging, as people with it appear to age far faster than normal. Using a new CRISPR/Cas9 gene therapy in a mouse model, they were able to slow down the pace of the condition, improve health, and increase lifespan.

What is Hutchinson-Gilford progeria?

Progeria is a degenerative disorder caused by a mutation in the LMNA gene. This disease has an early onset and progresses rapidly, and animals and humans with progeria show symptoms that are similar to regular aging, only on a much-accelerated timescale, giving them drastically shorter lifespans than normal. Humans with this condition rarely live very long, with the average being only 13 years old.

Continue reading “A CRISPR Gene Therapy for Hutchinson-Gilford Progeria” »

Hutchinson–Gilford progeria syndrome (HGPS) is a rare lethal genetic disorder characterized by symptoms reminiscent of accelerated aging. The major underlying genetic cause is a substitution mutation in the gene coding for lamin A, causing the production of a toxic isoform called progerin. Here we show that reduction of lamin A/progerin by a single-dose systemic administration of adeno-associated virus-delivered CRISPR–Cas9 components suppresses HGPS in a mouse model.

It’s inevitable in life, but aging isn’t really something people look forward to. Researchers have been seeking ways to reduce the impact of aging, not only because of vanity but also because as we age, there is a greater risk of certain serious health conditions like cancer, heart disease and neurodegenerative conditions like Alzheimer’s disease. Salk Institute scientists have now used CRISPR/Cas9, the gene-editing tool, to slow down aging. The work, reported in Nature Medicine, showed accelerated aging can be slowed in mice modeling a rare genetic disorder called Hutchinson-Gilford progeria syndrome.

“Aging is a complex process in which cells start to lose their functionality, so it is critical for us to find effective ways to study the molecular drivers of aging,” said the senior author of the report Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory. “Progeria is an ideal aging model because it allows us to devise an intervention, refine it and test it again quickly.”

Continue reading “Slowing the Aging Process” »

• Spherical nucleic acids are a class of personalized medicines for treating cancer and other diseases
• SNAs are challenging to optimize because their structures can vary in many ways
• Northwestern University team developed a library approach and machine learning to rapidly synthesize, analyze and select for potent SNA medicines

EVANSTON, Ill.— With their ability to treat a wide a variety of diseases, spherical nucleic acids (SNAs) are poised to revolutionize medicine. But before these digitally designed nanostructures can reach their full potential, researchers need to optimize their various components.

A Northwestern University team led by nanotechnology pioneer Chad A. Mirkin has developed a direct route to optimize these challenging particles, bringing them one step closer to becoming a viable treatment option for many forms of cancer, genetic diseases, neurological disorders and more.

LA JOLLA—(February 18, 2019) Aging is a leading risk factor for a number of debilitating conditions, including heart disease, cancer and Alzheimer’s disease, to name a few. This makes the need for anti-aging therapies all the more urgent. Now, Salk Institute researchers have developed a new gene therapy to help decelerate the aging process.

The findings, published on February 18, 2019 in the journal Nature Medicine, highlight a novel CRISPR/Cas9 genome-editing therapy that can suppress the accelerated aging observed in mice with Hutchinson-Gilford progeria syndrome, a rare genetic disorder that also afflicts humans. This treatment provides important insight into the molecular pathways involved in accelerated aging, as well as how to reduce toxic proteins via gene therapy.

“Aging is a complex process in which cells start to lose their functionality, so it is critical for us to find effective ways to study the molecular drivers of aging,” says Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory and senior author of the paper. “Progeria is an ideal aging model because it allows us to devise an intervention, refine it and test it again quickly.”

Continue reading “A new CRISPR/Cas9 therapy can suppress aging” »

There are 1.4 billion insects for each one of us. Though you often need a microscope to see them, insects are “the lever pullers of the world,” says David MacNeal, author of Bugged. They do everything from feeding us to cleaning up waste to generating $57 billion for the U.S. economy alone.

Today, many species are faced with extinction. When National Geographic caught up with MacNeal in Los Angeles, he explained why this would be catastrophic for life on Earth and why a genetically engineered bee could save hives—and our food supply—worldwide.