Lifeboat Foundation: Safeguarding Humanity
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Category: genetics

LONDON The world’s first life-saving gene therapy for children, developed by Italian scientists and GlaxoSmithKline, has been recommended for approval in Europe, boosting the pioneering technology to fix faulty genes.

The European Medicines Agency (EMA) said on Friday it had endorsed the therapy, called Strimvelis, for a tiny number of children with ADA Severe Combined Immune Deficiency (ADA-SCID) for whom no matching bone marrow donor is available.

Around 15 children a year are born in Europe with the ultra-rare genetic disorder, which leaves them unable to make a type of white blood cell. They rarely survive beyond two years unless their immune function is restored with a suitable bone marrow transplant.

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The duration of epigenetic responses underpinning transgenerational inheritance is determined by an active mechanism relying on the production of small RNAs and modulation of RNAi factors, dictating whether ancestral RNAi responses would be memorized or forgotten (credit: Leah Houri-Ze’evi et al./Cell)

According to epigenetics — the study of inheritable changes in gene expression not directly coded in our DNA — our life experiences may be passed on to our children and our children’s children. Studies on survivors of traumatic events have suggested that exposure to stress may indeed have lasting effects on subsequent generations.

But exactly how are these genetic “memories” passed on?

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1st of many steps in the gene editing oversight.

PRINCETON, N.J., March 29, 2016 /PRNewswire/ — WIRB-Copernicus Group® (WCG™), one of the world’s leading providers of solutions that measurably improve the quality and efficiency of clinical research, today announced that it has assembled a team of world-renowned experts to advise the company regarding the latest advances in gene therapy research. The WCG Gene Therapy™ Advisory Board will convene today in Princeton, NJ.

“Human gene therapy is one of the fastest-growing areas of medical research, and also one of the most promising,” said WCG Chairman and Chief Executive Officer Donald A. Deieso, Ph.D. “The advances made by scientists and clinicians in the field of gene therapy have enabled us to target disease at the genetic level, redefining the concept of precision medicine.” He added, “More than that, gene transfer researchers have succeeded – over the course of a single lifetime – in transforming the world’s most persistent and lethal viruses into disease-fighting allies in the quest to improve human health.”

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(Photo credit: AP Photo/Jack Plunkett, File)

Astro Teller is tired of the paranoia surrounding artificial intelligence and robotics. The famous computer scientist’s sensitivity around the topic may be understandable considering he bears the brunt of some of that skepticism as the head of X, the Alphabet (and formerly Google) moonshot factory working on many of the company’s futuristic AI and robotics projects.

This past weekend, Teller, whose official title is “captain of moonshots,” took to the stage at the inaugural Silicon Valley Comic Con hoping to dispel some of these misconceptions around AI. His physician wife, Danielle Teller, presented alongside him on some of the fear mongering associated with genetic engineering in humans. After their presentation, the Tellers sat down with FORBES to go deeper on the issue to explain what they hoped to accomplish with their talk.

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WIKIMEDIA, ROBINSON RCRISPR-a bacterial immune response best known for its genome-editing applications in the lab-has yet again been adapted for scientific purposes, this time to track RNA within cells. Considering the case of synapses — the proteins required for these neural connections are produced from RNAs located at these contacts.

“Just as CRISPR-Cas9 is making genetic engineering accessible to any scientist with access to basic equipment, RNA-targeted Cas9 may support countless other efforts for studying the role of RNA processing in disease or for identifying drugs that reverse defects in RNA processing”, study coauthor David Nelles of the University of California, San Diego, said in a press release. Defective RNA transport is linked to a host of conditions ranging from autism to cancer and researchers need ways to measure RNA movement in order to develop treatments for these conditions. “Our current work focuses on tracking the movement of RNA inside the cell, but future developments could enable researchers to measure other RNA features or advance therapeutic approaches to correct disease-causing RNA behaviors”. But, Gene Yeo, Associate Professor of Cellular and Molecular Medicine at UC San Diego, and his team have applied the technique as a flexible means to targeting RNA in live cells.

Jennifer Doudna, the creator of the CRISPR-Cas9 system for DNA editing, also works out of the University of California research system, and is listed as a co-author for this study. A guide RNA, along with the addition of an oligonucleotide sequence, sent the Cas9 RNA-ward.

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Scientists announced Thursday that they have built a single-celled organism that has just 473 genes — likely close to the minimum number of genes necessary to sustain its life. The development, they say, could eventually lead to new manufacturing methods.

Around 1995, a few top geneticists set out on a quest: to make an organism that had only the genes that were absolutely essential for its survival. A zero-frills life.

It was a heady time.

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This could have some truly profound implications for the treatment of all viruses, including HIV!

Researchers from Temple University have used the CRISPR/Cas9 gene editing tool to clear out the entire HIV-1 genome from a patient’s infected immune cells. It’s a remarkable achievement that could have profound implications for the treatment of AIDS and other retroviruses.

When we think about CRISPR/Cas9 we tend to think of it as a tool to eliminate heritable genetic diseases, or as a way to introduce new genes altogether. But as this new research shows, it also holds great promise as a means to eliminate viruses that have planted their nefarious genetic codes within host cells. This latest achievement now appears in Nature Scientific Reports.

Retroviruses, unlike regular run-of-the-mill viruses, insert copies of their genomes into host cells in order to replicate. Antiretroviral drugs have proven effective at controlling HIV after infection, but patients who stop taking these drugs suffer a quick relapse. Once treatment stops, the HIV reasserts itself, weakening the immune system, thus triggering the onset of acquired immune deficiency syndrome, or AIDS.

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Genetic mutations from extinct human relatives called the Denisovans might have influenced modern human immune systems, as well as fat and blood sugar levels, researchers say.

Very little is known about the Denisovans. The first evidence of them was discovered in Denisova Cave in Siberia in 2008, and DNA from their fossils suggests they shared an origin with Neanderthals but were nearly as genetically distinct from Neanderthals as Neanderthals were from modern humans.

Previous work found that any modern humans with ancestry outside of Africa inherited about 1.5 to 2.1 percent of their DNA from Neanderthals. In contrast, prior research suggested that substantial levels of Denisovan ancestry are found only in the Pacific islands of Melanesia. Scientists are increasingly uncovering the effects of Neanderthal ancestry on modern humans, from potential immune boosts to increased risks for depression, obesity, heart attacks, nicotine addiction. However, relatively little was known about the effects of Denisovan ancestry.

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On track to resolving defective RNA through CRISPR-Cas9.

According to a study published in journal Cell on March 17, researchers at University of California, San Diego School of Medicine, have found a way to track RNA in living cells. CRISPR-Cas9, a DNA-editing technique will be applied to target RNA in order to find cure for presently untreatable diseases such as cancer and autism.

There are many diseases that are associated with RNA behavior, which carries the genetic code from the cell’s nucleus. There was no technique found until now that could track RNA in living cells efficiently. However now, CRISPR-Cas9, which so far was only able to manipulate DNA, would now target RNA, which is also called RNA-targeted Cas9.

“Our current work focuses on tracking the movement of RNA inside the cell, but future developments could enable researchers to measure other RNA features or advance therapeutic approaches to correct disease-causing RNA behaviors”, said senior author Gene Yeo, PhD, associate professor of cellular and molecular medicine.

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