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Scientists at The University of Texas at Austin have redesigned a key component of a widely used CRISPR-based gene-editing tool, called Cas9, to be thousands of times less likely to target the wrong stretch of DNA while remaining just as efficient as the original version, making it potentially much safer.

Other labs have redesigned Cas9 to reduce off-target interactions, but so far, all these versions improve accuracy by sacrificing speed. SuperFi-Cas9, as this new version has been dubbed, is 4,000 times less likely to cut off-target sites but just as fast as naturally occurring Cas9. Bravo says you can think of the different lab-generated versions of Cas9 as different models of self-driving cars. Most models are really safe, but they have a top speed of 10 miles per hour.

“They’re safer than the naturally occurring Cas9, but it comes at a big cost: They’re going extremely slowly,” said Bravo. “SuperFi-Cas9 is like a self-driving car that has been engineered to be extremely safe, but it can still go at full speed.”

NOTE FROM TED: Research around aging discussed in this talk remains an ongoing field of study. Please do not look to this talk for health advice. TEDx events are independently organized by volunteers. The guidelines we give TEDx organizers are described in more detail here: http://storage.ted.com/tedx/manuals/tedx_content_guidelines.pdf.

Have you ever wondered how long you will live? And if so, how could you change that number to live drastically longer? The science might be in your favor: follow David Sinclair, Australian biologist and professor of genetics at Harvard University, as he shares his research on slowing and reversing the process of aging in mice, and how the same technology may someday be transferable to humans. David Sinclair, Australian biologist and professor of genetics at Harvard Universityhis insightful research into the science of age reversal and anti-aging medicine.

Continue reading “Is Aging Reversible? A Scientific Look with David Sinclair | David Sinclair | TEDxBoston” »

a cardiac arrhythmia disorder associated with sudden death in young adults.

The findings expand possibilities for predictive risk scoring and provide new targets for therapeutic study, according to Alfred George, Jr., MD, chair and the Alfred Newton Richards Professor of Pharmacology and a co-author of the study.

“Prior to this work, there were only two genomic regions associated with Brugada syndrome risk that were identified by genome-wide studies. Data from the new study greatly expands this to 12 regions with a total of 21 genetic signals to better explain risk for Brugada syndrome,” George said. “The results also provide the basis for a polygenic risk score that can be used to assess risk in individuals.”

A recent study published in Nature Genetics identified 10 new genetic regions associated with Brugada syndrome, a cardiac arrhythmia disorder associated with sudden death in young adults.

The findings expand possibilities for predictive risk scoring and provide new targets for therapeutic study, according to Alfred George, Jr., MD, chair and the Alfred Newton Richards Professor of Pharmacology and a co-author of the study.

“Prior to this work, there were only two genomic regions associated with Brugada syndrome risk that were identified by genome-wide studies. Data from the new study greatly expands this to 12 regions with a total of 21 genetic signals to better explain risk for Brugada syndrome,” George said. “The results also provide the basis for a polygenic risk score that can be used to assess risk in individuals.”

Scientists at Northwestern Medicine are using new advances in CRISPR gene-editing technology to uncover new biology that could lead to longer-lasting treatments and new therapeutic strategies for Human Immunodeficiency Virus (HIV).

The HIV epidemic has been overlooked during the COVID-19 pandemic but represents a critical and ongoing threat to human health with an estimated 1.5 million new infections in the last year alone.

Drug developers and research teams have been searching for cures and new treatment modalities for HIV for over 40 years but are limited by their understanding of how the virus establishes infection in the human body. How does this small, unassuming virus with only 12 proteins—and a genome only a third of the size of SARS-CoV-2—hijack the body’s cells to replicate and spread across systems?

Further study of newly-sequenced portions of the genome could also help scientists better understand how humans evolved particular traits, such as the bigger brains that sent them down a genetically distinct path from their great ape ancestors.

“The things that make our frontal cortex bigger come from the genes that map in these repetitive regions,” said Evan Eichler, a professor in the department of genome sciences at the University of Washington School of Medicine and also part of the research collaborative.

Advances in genomic sequencing technology could drive a renaissance of medical breakthroughs, the researchers say.

Scientists have managed to safely reverse the signs of aging in mice by genetically reprogramming some of their cells.

GABAergic dysfunctions have been implicated in the pathogenesis of schizophrenia, especially the associated cognitive impairments. The GABA synthetic enzyme glutamate decarboxylase 67-kDa isoform (GAD67) encoded by the GAD1 gene is downregulated in the brains of patients with schizophrenia. Furthermore, a patient with schizophrenia harboring a homozygous mutation of GAD1 has recently been discovered. However, it remains unclear whether loss of function of GAD1 leads to the symptoms observed in schizophrenia, including cognitive impairment. One of the obstacles faced in experimental studies to address this issue is the perinatal lethality of Gad1 knockout (KO) mice, which precluded characterization at the adult stage. In the present study, we successfully generated Gad1 KO rats using CRISPR/Cas9 genome editing technology.

Type 1 diabetes is caused by insulin deficiency resulting from immune-mediated destruction of pancreatic beta cells. Its cause is multifactorial, with complex genetic factors and a strong influence of certain HLA types that probably indicates T-cell-mediated disease.1 Despite attempts to halt the autoimmune destruction of the pancreatic islet at disease onset with immunotherapeutic approaches, 1 recovery of pancreatic beta-cell function remains a challenge because type 1 diabetes is not reversible, even early in the disease.

Correspondence from The New England Journal of Medicine — STAT1 Gain of Function, Type 1 Diabetes, and Reversal with JAK Inhibition.