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A novel technology for genome-editing a broad range of mutations in live organisms

The ability to edit genes in living organisms offers the opportunity to treat a plethora of inherited diseases. However, many types of gene-editing tools are unable to target critical areas of DNA, and creating such a technology has been difficult as living tissue contains diverse types of cells.

Now, Salk Institute researchers have developed a new tool—dubbed SATI—to edit the , enabling the team to target a broad range of mutations and cell types. The new genome-editing technology, described in Cell Research on August 23, 2019, could be expanded for use in a broad range of gene mutation conditions such as Huntington’s disease and the rare premature aging syndrome, progeria.

“This study has shown that SATI is a powerful tool for genome editing,” says Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory and senior author of the paper. “It could prove instrumental in developing effective strategies for target-gene replacement of many different types of mutations, and opens the door for using genome-editing tools to possibly cure a broad range of genetic diseases.”

The next trick for CRISPR is gene-editing pain away

The street performer was only 10 years old. He put knives through his arms and walked on hot embers. By 14 he was dead. Someone dared him to jump from a roof. He did it, knowing it wouldn’t hurt.

The case of the Pakistani boy with a rare genetic disorder was described in 2006. He could feel warmth and cold and the texture of objects. But he never felt pain.

Now scientists have paired the discovery with the gene-editing tool CRISPR, in what they say is a step toward a gene therapy that could block severe pain caused by diabetes, cancer, or car accidents without the addictive effects of opioids.

The photo points to the page for good reason

Both recent new Kindle books (will be paperbacks also in time) concern the two streams of this project. Primal Eye 1979–2019 outlines circuit designs and hard considerations and outlines MVT Posthuman Psychology. The other Kindle book — ZENET Game of Immortality — details some of the gaming and soft matters.


Everybody isn’t going to live forever even given new genetic techniques and improved medicines. When you reach a terminal state beyond medical science, the only options seem cryogenic preservation, actual death, or Artificial-Death. PRIMAL EYE 40 years on (1979 to 2019) includes Conscious Circuits, Artifical-Death and Posthuman Psychology.

Huge US government study to offer genetic counselling

But to generate the kind of long-term data set necessary for breakthroughs in precision medicine — which uses genomic, physiological and other data to tailor treatments to individuals — All of Us must retain these participants, ideally throughout their lives. That’s where genetic counselling comes in.


A firm hired by the National Institutes of Health will work with participants in a research programme that plans to sequence one million genomes.

‘Key player’ identified in genetic link to psychiatric conditions

Scientists have identified a specific gene they believe could be a key player in the changes in brain structure seen in several psychiatric conditions, such as schizophrenia and autism.

The team from Cardiff University’s Neuroscience and Mental Health Research Institute has found that the deletion of the gene CYFIP1 leads to thinning of the insulation that covers and is vital for the smooth and rapid communications between different parts of the .

The new findings, published in the journal Nature Communications and highlighted in the journal Nature Reviews Neuroscience, throws new light on the potential cause of and could ultimately point to new and more effective therapies.

University of California awarded new U.S. CRISPR-Cas9 patent

BERKELEY, Calif., Aug. 20, 2019 /PRNewswire/ — Today, the U.S. Patent and Trademark Office has awarded a new patent (U.S. 10,385,360) to the University of California (UC), University of Vienna, and Dr. Emmanuelle Charpentier covering nucleic acid molecules encoding single-molecule guide RNAs, as well as CRISPR-Cas9 compositions comprising single-molecule guide RNAs or nucleic acid molecules encoding single-molecule guide RNAs.

Over the past six months, UC’s U.S. CRISPR-Cas9 portfolio has sharply increased, and to date includes 11 separate patents for methods and compositions related to the gene-editing technology. Looking ahead, UC anticipates at least six additional related patents issuing in the near future, bringing UC’s total portfolio to 17 patents and spanning various compositions and methods including targeting and editing genes in any setting, such as within plant, animal, and human cells. The portfolio also includes patents related to the modulation of transcription.

“The USPTO has continually acknowledged the Doudna-Charpentier team’s groundbreaking work,” said Eldora L. Ellison, Ph.D., lead patent strategist on CRISPR-Cas9 matters for UC and a Director at Sterne, Kessler, Goldstein & Fox. “True to UC’s mission as a leading public university, the patent granted today and others in its CRISPR-Cas9 portfolio will be applied for the betterment of society.”

Long-Term Effects, Pathophysiological Mechanisms, and Risk Factors of Chemotherapy-Induced Peripheral Neuropathies: A Comprehensive Literature Review

Neurotoxic anticancer drugs, such as platinum-based anticancer drugs, taxanes, vinca alkaloids, and proteasome/angiogenesis inhibitors are responsible for chemotherapy-induced peripheral neuropathy (CIPN). The health consequences of CIPN remain worrying as it is associated with several comorbidities and affects a specific population of patients already impacted by cancer, a strong driver for declines in older adults. The purpose of this review is to present a comprehensive overview of the long-term effects of CIPN in cancer patients and survivors. Pathophysiological mechanisms and risk factors are also presented. Neurotoxic mechanisms leading to CIPNs are not yet fully understood but involve neuronopathy and/or axonopathy, mainly associated with DNA damage, oxidative stress, mitochondria toxicity, and ion channel remodeling in the neurons of the peripheral nervous system. Classical symptoms of CIPNs are peripheral neuropathy with a “stocking and glove” distribution characterized by sensory loss, paresthesia, dysesthesia and numbness, sometimes associated with neuropathic pain in the most serious cases. Several risk factors can promote CIPN as a function of the anticancer drug considered, such as cumulative dose, treatment duration, history of neuropathy, combination of therapies and genetic polymorphisms. CIPNs are frequent in cancer patients with an overall incidence of approximately 38% (possibly up to 90% of patients treated with oxaliplatin). Finally, the long-term reversibility of these CIPNs remain questionable, notably in the case of platinum-based anticancer drugs and taxanes, for which CIPN may last several years after the end of anticancer chemotherapies. These long-term effects are associated with comorbidities such as depression, insomnia, falls and decreases of health-related quality of life in cancer patients and survivors. However, it is noteworthy that these long-term effects remain poorly studied, and only limited data are available such as in the case of bortezomib and thalidomide-induced peripheral neuropathy.

Platinum-based anticancer drugs (i.e., cisplatin, oxaliplatin), proteasome/angiogenesis inhibitors (bortezomib/thalidomide), vinca alkaloids (i.e., vincristine, vinorelbine) and taxanes (i.e., paclitaxel, docetaxel) are the most common anticancer drugs used as first-line chemotherapy for several cancers, including colorectal, gastric, breast and lung cancers, and multiple myeloma. Despite their different action mechanisms, all these anticancer drugs share a common adverse and disabling effect for patients, namely CIPN (Balayssac et al., 2011). CIPN has a considerable impact on cancer treatments and their related symptoms severely affect patients’ daily activities and quality of life. Thus CIPN is often the main adverse effect leading to the reduction or discontinuation of chemotherapy.

News of Note—Changing multiple genes with CRISPR; Accelerating recovery after chemo and radiation

The evolving gene-editing technology CRISPR-Cas9 is useful for changing one gene, or maybe a few genes at a time. A team at ETH Zurich has tweaked the technology so they can change 25 different gene sites at once. Instead of using the Cas9 enzyme to do the DNA cutting, though, they used Cas12a. That allowed them to create a long “address list” of gene sites to target, they explained in the journal Nature Methods. They created a DNA molecule called a plasmid to store the list, inserted it in human cells and were able to modify several genes, they reported. (Release)

Chemotherapy and radiation suppress blood stem cells, often for several weeks or even months after cancer treatments are complete. This leaves patients vulnerable to infections and other health problems. Scientists at the University of California, Los Angeles have created a new drug that targets the protein tyrosine phosphatase-sigma (PTP-sigma), which is prevalent on blood stem cells. They showed that blocking the protein in rodent models with the drug, called DJ009, helped blood cells recover more quickly after they were damaged by radiation. They published their findings in the journal Nature Communications. (Release)

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