Genetic Engineering in the Press by GEG
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Small molecules set up security system to defend the genome: Thousands of RNA molecules use a search algorithm to identify and silence foreign genetic sequences in cells

Small molecules set up security system to defend the genome: Thousands of RNA molecules use a search algorithm to identify and silence foreign genetic sequences in cells | Genetic Engineering in the Press by GEG | Scoop.it
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Thousands of short RNA molecules with diverse genetic sequences serve as security guards to identify and silence attempts to invade the genome, such as DNA inserted by viruses or parasitic elements known as transposons.
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We won’t use CRISPR to make super-smart babies—but only because we can’t

We won’t use CRISPR to make super-smart babies—but only because we can’t | Genetic Engineering in the Press by GEG | Scoop.it
After the Storm — A Responsible Path for Genome Editing
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Gene experts speculate that our worst gene-editing fears won’t come true because they are too complex for us to pull them off.

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FDA grants accelerated review for CRISPR's gene therapy CTX001 for SCD - CRISPR Therapeutics 

FDA grants accelerated review for CRISPR's gene therapy CTX001 for SCD - CRISPR Therapeutics  | Genetic Engineering in the Press by GEG | Scoop.it
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CRISPR Therapeutics (NASDAQ:CRSP) and collaboration partner Vertex Pharmaceuticals (NASDAQ:VRTXannounce that the FDA has granted Fast Track status for gene therapy CTX001 for the treatment of sickle cell disease (SCD).

Fast Track provides for more frequent interaction with the FDA review team and a rolling review of the marketing application.

CTX001 is a gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.

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Jiankui He Vortrag zu den CRISPR-Babies - YouTube

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Chinese scientist He Jiankui, who claimed to have helped make the world's first gene-edited babies, said he was “proud” of his work when speaking at a genome conference in Hong Kong on Wednesday. He claimed his widely-criticized research aimed at helping children resist possible future HIV contraction. (Video  of Second International Summit on Human Genome Editing)

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UK’s cell and gene therapy industry continues to strengthen 

UK’s cell and gene therapy industry continues to strengthen  | Genetic Engineering in the Press by GEG | Scoop.it
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The UK’s cell and gene therapy industry has seen a 60% increase in manufacturing space in the last year, new research published by the Cell and Gene Therapy (CGT) Catapult has revealed.

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Penn receives $10.7 million to advance CAR-T therapy research into solid tumors | The Daily Pennsylvanian

Although CAR-T cells have been revolutionized the treatment of leukemia and bone marrow cancers, researchers have not yet had the same success in treating solid tumors like lung cancer.  
BigField GEG Tech's insight:

Penn’s Translational Center of Excellence for Lung Cancer Immunology at the Abramson Cancer Center was awarded a $10.7 million grant on Oct. 22 to support the advancement of research for CAR-T therapy in lung cancer patients.

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New CRISPR tool opens up more of the genome for editing

New CRISPR tool opens up more of the genome for editing | Genetic Engineering in the Press by GEG | Scoop.it
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MIT researchers have identified a new CRISPR enzyme that can target almost half of the genome’s “zipcodes.” The advance may make it possible to use the CRISPR system to edit many more disease-specific mutations.
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FDA lifts clinical hold of Vertex/CRISPR Therapeutics’ sickle cell drug | FierceBiotech

FDA lifts clinical hold of Vertex/CRISPR Therapeutics’ sickle cell drug | FierceBiotech | Genetic Engineering in the Press by GEG | Scoop.it
BigField GEG Tech's insight:
The FDA lifted a clinical hold it placed on Vertex and CRISPR Therapeutics’ gene-edited stem cell therapy for sickle cell disease. The companies are on track to start a human study in sickle cell by the end of the year.
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Japan Set to Allow Gene Editing in Human Embryos

Japan Set to Allow Gene Editing in Human Embryos | Genetic Engineering in the Press by GEG | Scoop.it
Scientific American is the essential guide to the most awe-inspiring advances in science and technology, explaining how they change our understanding of the world and shape our lives.
BigField GEG Tech's insight:

Japan has issued draft guidelines that allow the use of gene-editing tools in human embryos. The proposal was released by an expert panel representing the country’s health and science ministries on 28 September.

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Nobel Prize in medicine goes to cancer immunotherapy researchers - 

Nobel Prize in medicine goes to cancer immunotherapy researchers -  | Genetic Engineering in the Press by GEG | Scoop.it
James P. Allison and Tasuku Honjo, from the United States and Japan, win prestigious award
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The Nobel Prize in physiology or medicine was awarded Monday to cancer researchers James P. Allison and Tasuku Honjo whose studies led to the development of drugs that unleash the human immune system against the deadly disease.

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Decoding the structure of an RNA-based CRISPR system

LA JOLLA—Over the past several years, CRISPR-Cas9 has moved beyond the lab bench and into the public zeitgeist. This gene-editing tool holds promise for correcting defects inside individual cells and potentially healing or preventing many human ailments. But the Cas9 system alters DNA, not RNA, and some experts believe that being able to modify RNA ultimately may prove just as useful.
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Now scientists from the Salk Institute are reporting for the first time the detailed molecular structure of CRISPR-Cas13d, a promising enzyme for emerging RNA-editing technology. They were able to visualize the enzyme thanks to cryo-electron microscopy (cryo-EM), a cutting-edge technology that enables researchers to capture the structure of complex molecules in unprecedented detail. The findings were reported September 20, 2018, in the journal Cell.

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Massachusetts Institute of Technology's Broad Institute gets to keep its Crispr patent, says US appeals court —

Massachusetts Institute of Technology's Broad Institute gets to keep its Crispr patent, says US appeals court — | Genetic Engineering in the Press by GEG | Scoop.it
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A federal appears court voted to uphold patents filed by scientists at MIT and Harvard, saying that they were non-obvious compared to those filed earlier by another team.

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CNW | Novartis receives Health Canada approval of its CAR-T cell therapy, Kymriah™ 

CNW | Novartis receives Health Canada approval of its CAR-T cell therapy, Kymriah™  | Genetic Engineering in the Press by GEG | Scoop.it
DORVAL, QC, Sept. 6, 2018- Novartis receives Health Canada approval of its CAR-T cell therapy, Kymriah™ (tisagenlecleucel)i.
BigField GEG Tech's insight:

Health Canada has approved Kymriah for use in pediatric and young adult patients 3 to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) who are refractory, have relapsed after allogenic stem cell transplant (SCT) or are otherwise ineligible for SCT, or have experienced second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, high grade B-cell lymphoma and DLBCL arising from follicular lymphomai.

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Alnylam Receives Approval of ONPATTRO™ (patisiran) in Europe 

Alnylam Receives Approval of ONPATTRO™ (patisiran) in Europe  | Genetic Engineering in the Press by GEG | Scoop.it
BigField GEG Tech's insight:

Alnylam Pharmaceuticals, Inc.(Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the European Commission (EC) has granted marketing authorization for ONPATTRO™(patisiran) for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or stage 2 polyneuropathy. ONPATTRO is based on Nobel Prize-winning science and is the first-ever RNA interference (RNAi) therapeutic to be approved in the European Union.

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Chinese Scientist Who Claimed to Make Genetically Edited Babies Is Kept Under Guard - The New York Times

Chinese Scientist Who Claimed to Make Genetically Edited Babies Is Kept Under Guard - The New York Times | Genetic Engineering in the Press by GEG | Scoop.it
He Jiankui, who was last seen in public in November soon after his stunning announcement, is sequestered in a guesthouse in the city of Shenzhen.
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First children set to receive Novartis’ Kymriah - PharmaTimes

First children set to receive Novartis’ Kymriah - PharmaTimes | Genetic Engineering in the Press by GEG | Scoop.it
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The first children to receive Novartis’ Kymriah – a ‘game-changing’ CAR-T therapy for cancer - will start treatment at Great Ormond Street Hospital in London this week, NHS England has announced. - News - PharmaTimes

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CRISPR scientist in China claims his team’s research has resulted in the world’s first gene-edited babies

CRISPR scientist in China claims his team’s research has resulted in the world’s first gene-edited babies | Genetic Engineering in the Press by GEG | Scoop.it
Update: The story is getting even more convoluted. When contacted by TechCrunch’s Rita Liao, a representative at the hospital that reportedly approved Jiankui He’s study stated “what we can say for sure is that the gene editing process did not take place at our hospital. The babie…
BigField GEG Tech's insight:

In what would represent a dramatic and ethically fraught escalation of CRISPR research, a scientist from a university in Shenzhen, China claims he has succeeded in helping create the world’s first genetically-edited babies. Jiankui He told the Associated Press that twin girls were born earlier this month after he edited their embryos using CRISPR technology to remove the CCR5 gene, which plays a critical role in enabling many forms of the HIV virus to infect cells.

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DefiniGEN Licenses CRISPR-Cas9 Gene Editing Technology to Develop Cell Models for Optimized Metabolic Disease Drug Development 

DefiniGEN Licenses CRISPR-Cas9 Gene Editing Technology to Develop Cell Models for Optimized Metabolic Disease Drug Development  | Genetic Engineering in the Press by GEG | Scoop.it
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DefiniGEN Ltd has announced the commercial licensing of CRISPR-Cas9 gene-editing technology from Broad Institute of MIT and Harvard in the USA, to develop human cell disease models to support preclinical metabolic disease therapeutic programs.

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Pre-Existing Immunity to CRISPR Found in 96% of People in Study

Pre-Existing Immunity to CRISPR Found in 96% of People in Study | Genetic Engineering in the Press by GEG | Scoop.it
The immune systems of a large majority of people could already be primed to attack and possibly even disable a key component of CRISPR-Cas9 gene editing
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Recent paper, published in the peer-reviewed journal Nature Medicine, suggests those results from January weren’t a fluke. In the latest research, a team of scientists in Germany exposed blood samples from 48 healthy volunteers to Cas9 (a DNA-cutting enzyme) derived from a bacterium called Streptococcus pyogenes. (Cas9 from S. pyogenes is one of the most common DNA-cutting enzymes used in CRISPR R&D, if not the most commonly used.)

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Use the patent system to regulate gene editing - Nature

Use the patent system to regulate gene editing - Nature | Genetic Engineering in the Press by GEG | Scoop.it
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Governments should use patents to shape the deployment of CRISPR–Cas9 as they have done for past technologies, argues Shobita Parthasarathy.

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Agricultural research, or a new bioweapon system? - Science

Agricultural research, or a new bioweapon system? - Science | Genetic Engineering in the Press by GEG | Scoop.it

https://www.darpa.mil/program/insect-allies

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The Pentagon is studying whether insects can be enlisted to combat crop loss during agricultural emergencies. The bugs would carry genetically engineered viruses that could be deployed rapidly if critical crops such as corn or wheat became vulnerable to a drought, a natural blight or a sudden attack by a biological weapon. The concept envisions the viruses making genetic modifications that protect the plants immediately, during a single growing season.

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Penn Discovers New, Rare Mechanism for ALL to Relapse after CAR T Cell Therapy – PR News

Induction of resistance to chimeric antigen receptor T cell therapy by transduction of a single leukemic B cell
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A single leukemia cell, unknowingly engineered with the leukemia-targeting chimeric antigen receptor (CAR) lentivirus and infused back into a patient, was able to reproduce and cause a deadly recurrence of pediatric B-cell acute lymphoblastic leukemia (ALL).

Nature Medicine published the findings.

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Gene editing could eliminate mosquitoes, but is it a good idea?

Gene editing could eliminate mosquitoes, but is it a good idea? | Genetic Engineering in the Press by GEG | Scoop.it

In the study, scientists at Imperial College London used the technology to wipe out a population of caged mosquitoes that are able to transmit malaria, targeting a genetic sequence that leads to male and female traits. After a number of generations, they found that 100% of these mosquitoes were affected.

 

https://www.nature.com/articles/nbt.4245

 

BigField GEG Tech's insight:

Researchers have rendered a population of mosquitoes in a lab sterile using the gene-editing tool CRISPR-Cas9 by homing in on a specific target in insect DNA -- the doublesex gene -- raising the possibility of eradicating disease-carrying species of the insect entirely, according to a new study published Monday in Nature Biotechnology.

But even when it comes to suppressing or extinguishing a species of mosquito, are we opening Pandora's Box and releasing something into the world we don't fully understand? Or will the only major impact be a positive one for humans while other organisms -- less harmful ones, ideally -- take these bugs' place in the ecosystem?

 

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'Switchable' CAR T-cell Therapy Kills Advanced Pancreatic Cancer Cells in Mice, Study Reports

'Switchable' CAR T-cell Therapy Kills Advanced Pancreatic Cancer Cells in Mice, Study Reports | Genetic Engineering in the Press by GEG | Scoop.it
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A “switchable” CAR T-cell immunotherapy was able to completely eliminate cancer in mice whose tumors came from cells of people with advanced pancreatic cancer, including cancer cells that had moved to distant organs, a new study reports.

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CRISPR Pioneer Jennifer Doudna Opens Lab at Bay Area’s Gladstone Institutes

CRISPR Pioneer Jennifer Doudna Opens Lab at Bay Area’s Gladstone Institutes | Genetic Engineering in the Press by GEG | Scoop.it
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CRISPR pioneer Jennifer Doudna has set up shop in San Francisco’s Gladstone Institutes. Doudna, a UC Berkley professor credited with the co-discovery of CRISPR-Cas9 genome editing technique, will focus on the development of new ways to implement the gene-editing technique for disease treatment.

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Novartis, Gilead CAR-T therapies approved in Europe, putting spotlight on global manufacturing

Novartis, Gilead CAR-T therapies approved in Europe, putting spotlight on global manufacturing | Genetic Engineering in the Press by GEG | Scoop.it
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Kymriah and Yescarta are churned out in a matter of weeks — a production task that gets more difficult as approvals expand their geographic reach.

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