Bold Chinese Experiment Genetically Engineers Monkeys, maybe makes them Smarter, definitely raises some ethical questions

Chinese researchers are going hard lately! Following projects that include genetically modifying embryos and letting two develop into twin human babies, and cloning primates, another envelope-pushing report comes from the Chinese Bio-Science community—this time by inserting a human version of a gene into a Rhesus Monkey. The gene MCPH1 is thought to play an important role in human brain development and contribute to the distinctively human cognitive ability. The genetically modified monkeys exhibited slower (more human-like) brain development and possibly even improved cognitive ability. This work was published by Oxford University Press on behalf of China Science Publishing & Media Ltd., which is ostensibly a peer-review journal, but not PLOS or PNAS, and it unclear if this work would be given the green light at an American University. There will certainly be debate in the press about this topic, which should be thrilling, but hopefully it will hasten a some thoughtful conclusions.

Read the original article HERE and other summaries here and here and here.

Colorado-based Gene-Editing Company gets Series C Financing

Inscripta Completes $20M Expansion of Series C Financing, Increasing Total Round to $105.5M

BOULDER, Colo.--(BUSINESS WIRE)--Inscripta, a leading gene editing technology company, today announced that it has increased its previously announced Series C financing with an additional $20 million from existing investors. The new funding adds to the $85.5 million financing Inscripta announced in 2018, bringing the total raised for the round to $105.5 million.

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A bioengineer, a biopunk, and a biotech reporter talk about Genetic Engineering

The Future of Genetic Engineering

A bioengineer, a biopunk, and a biotech reporter square off onstage about our neobiological future.

By Jane Metcalfe

In a new documentary that is still in production, filmmaker Cory Sheehy follows renowned bioengineer George Church and biotech reporter Antonio Regalado to China for the 2nd International Summit on Human Genome Editing. That’s where biophysicist He Jiankui made the stunning announcement that he had edited the DNA of two twin girls who were born in November 2018. Back in California, the filmmaker catches up with biohacker Josiah Zayner, whose attention-grabbing exploits—part protest, part performance art—include injecting himself with a CRISPR-Cas9 plasmid.

Watch the video here …

Gene-Edited Anolis Lizards

UGA scientists create world’s first gene-edited lizards

A group of University of Georgia researchers led by geneticist Douglas Menke has become the first in the world to successfully produce a genetically modified reptile—specifically, four albino lizards—using the CRISPR-Cas9 gene-editing tool. The team’s results, which appeared online March 31, have been submitted for peer review.

“Reptiles are very understudied in terms of their reproductive biology and embryonic development,” said Menke, associate professor in the department of genetics. “There are no good methods to manipulate embryos like we can easily do with mammals, fish or amphibians. To our knowledge, no other lab in the world has produced a genetically altered reptile.”

Gene manipulation using CRISPR typically involves injecting gene-editing solutions into an animal’s newly fertilized egg or single-cell embryo, causing a mutation in the DNA that is reproduced in all subsequent cells. However female reptiles can store sperm in their oviducts for long periods, making it difficult to pinpoint the exact moment of fertilization. Also, the physiology of their fertilized eggs, which have pliable shells with no air space inside, presents challenges for manipulating embryos without damaging them.

Working with the species Anolis sagrei, commonly called the brown anole, Menke’s team overcame these challenges by microinjecting CRISPR proteins into multiple immature eggs, or oocytes, still located in the lizards’ ovaries. Targeting the tyrosinase gene, they successfully injected 146 oocytes from 21 lizards, then waited for the oocytes to be fertilized naturally. Within a few weeks, they realized their goal: four offspring displaying the telltale trait of albinism, which results when tyrosinase is inactivated.

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CRISPR cuts 13,000 times in one cell

Genome engineers made more than 13,000 CRISPR edits in a single cell

Rewriting Life

A team at George Church’s Harvard lab wants to redesign species with large-scale DNA changes.

by Antonio Regalado (MIT Technology Review)

Since its invention, CRISPR has let scientists introduce DNA changes at specific locations in a genome. Often these precise changes are made one at a time.

Perhaps not for much longer. A team at Harvard University says it has used the technique to make 13,200 genetic alterations to a single cell, a record for the gene-editing technology.

The group, led by gene technologist George Church, wants to rewrite genomes at a far larger scale than has currently been possible, something it says could ultimately lead to the “radical redesign” of species—even humans. Large-scale gene editing of this sort has been tried before. In 2017, an Australian team led by Paul Thomas peppered the Y chromosome of mice with edits and succeeded in blasting it out of existence. That strategy is being eyed as a potential treatment for Down syndrome, a genetic disorder caused by an extra chromosome.

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Is CRISPR a National Security Threat?

National Security Implications of Gene Editing

“CRISPR-Cas9 is here and now, he pointed out. “Gene editing has a number of near term and longer term ramifications that … have implications for national security, intelligence and defense,” he said.

If the research goes clandestine, the technology could be used to modify various physiological functions in humans both before birth and perhaps key operational points or optimized points after birth, he said.

With genomic knowledge of weaknesses and susceptibilities to certain diseases, populations could be targeted in order to cause instability in societies.”

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FDA slows down CRISPR

Gene Editing Promise Stalled at FDA

The FDA is currently claiming regulatory authority over gene-edited livestock based on a decades-old administrative decision related to older transgenic biotechnology, where a foreign gene is inserted into an animal’s genome. Gene editing is making simple changes or deletions within an animal’s native genome.

NPPC is pushing for regulatory oversight by the USDA. The USDA has already established the right regulatory framework by adopting a risk-based approach to reviewing potential genetic changes in plants. It easily could adapt that approach for livestock and regulate gene-edited animals under the Animal Health Protection Act. 

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One-stop shopping for your gene-editing needs

Brand new CRISPR/Cas9 Platform: One-stop Gene Knockout/knockin Service for Precise Gene Editing

New York, Mar 25th, 2019 - Creative Biogene, as a leading provider focuses on offering professional products and services to accelerate gene research, recently announces the brand new CRISPR/Cas9 Platform, which provides comprehensive gene editing services and products. With talented and experienced scientists, Creative Biogene can offer more reliable and professional service to support gene editing projects.

Read the whole press release here …

Chinese gene editing therapies are developing

China’s race to test ‘mutation-free’ gene-editing technology on cancer patients

BEIJING — China could be just over a year away from clinical trials of a new gene-editing therapy with an unprecedented high level of safety, according to a team of Chinese scientists involved in the research programme.

The scientists said the research, based on groundbreaking work published in the journal Science earlier this month, could help save the lives of many patients battling deadly diseases including cancer.

The existing genome-editing method works like a shotgun, breaking up a large numbers of genome strands and sometimes missing its intended target, causing unnecessary damage to cells.

The new tool under development in China targets and swaps individual “letters” in the DNA with extreme precision, avoiding cuts to the strands and significantly reducing the risk of unexpected mutations.

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Bloomberg covers important investor interest in gene editing therapyBloomberg covers important investor interest in gene editing therapy

Biotech Investors Zero In on Gene Therapy as Next Big Frontier

(Bloomberg) -- Biotech investors are bullish on the market for gene therapies, even though actual sales of a few existing drugs have so far failed to impress some Wall Street analysts. Fund managers and venture capitalists discussed their outlook for the technology in interviews in Boston, where investors gathered for the Cowen health-care conference. Investor interest in gene-therapy stocks is high, as investing has paid off this year. Roche Holding AG and Biogen Inc. recently snapped up a pair of drug developers, boosting speculation of more deals. Also, U.S. regulators say they expect to approve 10 to 20 new medicines by 2025, while insurers grapple with treatment costs that can run into the hundreds of thousands of dollars.

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National Academies on the Call for Moratorium on and International Governance of Heritable Gene Editing

Statement on Call for Moratorium on and International Governance Framework for Clinical Uses of Heritable Genome Editing

“A commentary published in Nature calls for a moratorium on clinical uses of heritable human genome editing and the establishment of an international governance framework. (We responded to the commentary with a joint letter, which is an abbreviated version of this statement.) The call comes following claims by a scientist in China to have edited the genes of early embryos, in treatments that resulted in the birth of twins. The scientist’s work – revealed at the Second International Summit on Human Genome Editing in Hong Kong, jointly organized by our Academies, was condemned by the summit organizers and by much of the wider scientific community.”

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Increasing CRISPR efficiency, and specificity with zinc-finger proteins (original article)

Diversifying the structure of zinc finger nucleases for high-precision genome editing

Abstract—Genome editing for therapeutic applications often requires cleavage within a narrow sequence window. Here, to enable such high-precision targeting with zinc-finger nucleases (ZFNs), we have developed an expanded set of architectures that collectively increase the configurational options available for design by a factor of 64. These new architectures feature the functional attachment of the FokI cleavage domain to the amino terminus of one or both zinc-finger proteins (ZFPs) in the ZFN dimer, as well as the option to skip bases between the target triplets of otherwise adjacent fingers in each zinc-finger array. Using our new architectures, we demonstrate targeting of an arbitrarily chosen 28 bp genomic locus at a density that approaches 1.0 (i.e., efficient ZFNs available for targeting almost every base step). We show that these new architectures may be used for targeting three loci of therapeutic significance with a high degree of precision, efficiency, and specificity.

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CRISPR-based gene therapy, speculating about a time-table

We’re still a long way from using gene-editing for medical conditions?

Gene editing has been in the news lately due to an ethically reckless experiment in which human embryos were subjected to an inefficient form of gene editing. The subjects, now born, gained uncertain protection from HIV in exchange for a big collection of potential risks. A large number of ethicists and scientists agreed that this isn't the sort of thing we should be using gene editing for.

Gene editing will likely always come with a bit of risk; when you're cutting and pasting DNA in millions of cells, extremely rare events can't be avoided. So the ethical questions come down to how we can minimize those risks and what conditions make them worth taking.

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Crispr Therapeutics AG has treated its first human

Crispr Infuses First Human in Landmark Gene-Editing Study

Crispr Therapeutics AG  shares surged after the company said it has treated the first human with the same genetic technology that shares its name in an early-stage study.

Crispr Therapeutics and partner Vertex Pharmaceuticals Inc. said on Monday morning that the first patient in a trial using CTX001, a therapy created using Crispr technology, as a treatment for the rare blood disease, beta thalassemia, received the one-time medicine. The pair also announced the enrollment of the first patient has started in a parallel study for the medicine in sickle-cell disease with the first dosing on track for mid-year.

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CRISPR Gene Editing advances

Gene Editing is trickier THAN expected—BUT fixes are in sight

Of all the big, world-remaking bets on the genome-editing tool known as Crispr, perhaps none is more tantalizing than its potential to edit some of humanity’s worst diseases right out of the history books. Just this week, Crispr Therapeutics announced it had begun treating patients with an inherited blood disorder called beta thalassemia, in the Western drug industry’s first test of the technology for genetic disease. But despite the progress, there remain a host of unknowns standing in the way of Crispr-based medicines going mainstream, chief among them safety.

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World Health Organization might make some human genome editing rules

Human genome editing is here – now we have to decide who is in charge

The World Health Organization (WHO) will convene a meeting this month to develop global standards of governance for human genome editing. This is a welcome move. Although the committee has no powers to enforce compliance – it is still a matter for individual nations to decide on regulations, with China reportedly updating its rules earlier this week – the WHO committee’s recommendations will be influential and far-reaching in their ambition. But I hope committee members will bear a few points in mind in their discussions.

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Genome editing and global markets overview

Genome Editing: Technologies and Global Markets

Report Scope:
This study is focused on the market side of genome editing and provides a comprehensive review of genome editing technologies, along with updates on the latest related progress in the field. Different market segments for this specific market are covered.

Download the full report: https://www.reportbuyer.com/product/3756560 

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After Scandal, China Proposes New Rules for Gene Editing

China has proposed new rules for gene editing and other “high risk” biomedical technologies. The proposals come three months after a Chinese researcher was widely criticized after claiming he had created the world’s first genetically-edited babies.

A draft of the measures was published this week on the website of China’s National Health Commission. The new rules would cover gene editing, stem cell experiments and biological products created for the human body, the Chinese government-supported Global Times newspaper reported.

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Chinese scientists have cloned genetically altered, non-Human, primates too

“This time last year, the first primates cloned through a nucleus transfer technique made headlines around the world. Now, Chinese researchers have pushed the envelope even further – by breaking a regulatory gene in macaques before cloning them. According to the researchers, cloning genetically altered primates has clear benefits for medical testing. But in the wake of controversy over gene editing on humans, progress in this contentious area could be outpacing ethics.

After the new experiment, five infant macaques born at the Chinese Academy of Sciences' Institute of Neuroscience in Shanghai all share the exact same genes, derived from a fibroblast taken from the skin of a donor monkey. More importantly, they all carry a copy of one specific gene – a version of BMAL1 that had been altered in the donor using CRISPR/Cas9 gene-editing technology.”

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