CRISPR corrects Duchenne muscular dystrophy mutation

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Abstract

Mutations in the dystrophin gene cause Duchenne muscular dystrophy (DMD), which is characterized by lethal degeneration of cardiac and skeletal muscles. Mutations that delete exon 44 of the dystrophin gene represent one of the most common causes of DMD and can be corrected in ~12% of patients by editing surrounding exons, which restores the dystrophin open reading frame. Here, we present a simple and efficient strategy for correction of exon 44 deletion mutations by CRISPR-Cas9 gene editing in cardiomyocytes obtained from patient-derived induced pluripotent stem cells and in a new mouse model harboring the same deletion mutation. Using AAV9 encoding Cas9 and single guide RNAs, we also demonstrate the importance of the dosages of these gene editing components for optimal gene correction in vivo. Our findings represent a significant step toward possible clinical application of gene editing for correction of DMD.

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Boston Herald gives a short intro to genetic testing

Gene mutations alert patients to cancer risks

Genetic testing that alerts patients to harmful gene mutations is paving the way to aid in preventing inherited cancers.

Cape Cod natives and sisters Liz Ellis, Katie Paquin and Christine Leary, who were all diagnosed with breast cancer within one year, carry the PALB2 gene, which is found to increase the risk of breast cancer.

Dr. Michael Misialek, associate chair of pathology at Newton-Wellesley Hospital, said knowing about a family history of cancer is the first step in taking preventative measures against the disease and getting tested for harmful genetic mutations.

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Massive family tree predicts life span is 16% heritable

Thirteen million degrees of Kevin Bacon: World’s largest family tree shines light on life span, who marries whom

Researchers have published what may be the validated largest family tree ever: a genealogy database stretching back 5 centuries that links 13 million people related by blood or marriage. The tree has already led to such insights as the link between genes and longevity and why our ancestors married whom they did. And researchers say that’s just a start.

“This study is an impressive and clever use of crowdsourcing data to address a number of interesting scientific questions,” says geneticist Peter Visscher of the University of Queensland in Brisbane, Australia, who was not involved with the work. The tree’s bigger promise, he and others say, could come if it were linked to health information to explore the role of genetics in diseases.

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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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Cancer immunotherapy improved with CRISPR

Researchers engineer immune cells to fight cancer

Deep in the cells of the human immune system, DNA is constantly being replicated, transcribed and even mutated — but rarely does it change dramatically. Like every other living organism, humans and their genes developed from millions of years of evolutionary pruning.

But to Yale microbiologists, altering the entire genomes of T-cells — the body’s main offensive weapon against diseases such as cancer — is as simple as putting together a Lego set.

In a new study published in the journal Nature Methods on Feb. 25, researchers at the Sidi Chen Lab at Yale have come up with a new way to use the gene-editing technology CRISPR that significantly improves the technology’s efficiency. By allowing scientists to select multiple genes to include in the same CRISPR system, scientists will now be able to edit their samples’ genomes in one go, saving time and money in the process. These findings have considerable promise for engineering T-cells that can fight off cancers such as leukemia and lymphoma.

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Potential paths to an HIV cure

Second-Ever HIV Cure Seen In London AIDS Patient Is Rare, But It Might Not Have To Be

For just the second time, a patient with AIDS appears to have been cured of the disease after receiving a stem cell transplant from a donor with a genetic mutation that provides resistance to HIV, which causes AIDS. But the mutation is exceedingly rare, raising controversial questions of whether it may be replicated using nascent gene-editing tools like CRISPR/Cas9.

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Overlooked potential in Immunochemicals Market?

Global Immunochemicals Market to Witness Exponential Rise in Revenue Share During the Forecast Period

Immunochemistry is a branch of science that deals with the study of the immune system. Immunochemical methods do not require extensive and destructive sample preparation and expensive instrumentation. Most immunochemical methods are based on simple photo, fluoro or luminometric detection. These methods have rapidly replaced chromatographic techniques in clinical diagnostics and offer fast detection of antibodies associated with specific diseases, hormones, disease biomarkers, and pharmaceuticals. Most commonly used assays in clinical immunochemistry involve either quantitative or qualitative formats using enzyme-linked immunosorbent assays (ELISAs), immunochromatography in the form of lateral-flow devices like dip-sticks and test strips, and Western Blot assays used to interpret data from protein analysis with gel electrophoresis. Moreover, it is also used in food safety assessment, environmental analysis, and the target chemical structures cover the widest range of molecular weight approximately hundreds of kilo Dalton (kD). Immunochemicals also develop antibody assays for cholera, anthrax, and diphtheria toxins.

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Protein-Lipid twist for new antiviral therapies

Team discovers protein, lipid connection that could aid new influenza therapies

The connection between an influenza virus surface protein and a host cell lipid has been discovered by researchers at the University of Maine and the National Institutes of Health. Confirmation of direct interaction between the protein and lipid could lead to new antiviral therapies.

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Informed speculation regarding upcoming Epigenetics Markets

Epigenetics Market 2019-2025 By (Growth Potential, Opportunities, Drivers, Industry-Specific Challenges And Risks)

Report Titled “Epigenetics Market Exploration Report Forecast 2019-2025” includes a comprehensive study of the important sections to provide insights on the Epigenetics Market dynamics till 2025, which would enable the stakeholders to capitalize on prevailing market opportunities, newest industry data and Epigenetics Industry future trends, allowing you to identify the products and end users driving Revenue growth and effectiveness.

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Science covers CRISPR in crop plants

Corn and other important crops can now be gene edited by pollen carrying CRISPR

The genome editor CRISPR has transformed many areas of biology, but using this tool to enhance certain varieties of crops such as wheat and corn remains difficult because of the plants’ tough cell walls. Now, a major agricultural company has creatively solved that problem by using pollen from one genetically modified plant to carry CRISPR’s components into another plant’s cells. The solution promises to speed the creation of better and more versatile crops, scientists say.

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CRISPR show promise with progeria syndrome mice

CRISPR-Cas9 Therapy May Suppress Aging

CRISPR-Cas9 genome editing therapy has been shown by the Salk Institute team to suppress the accelerated aging observed in mice with Hutchinson-Gilford Progeria syndrome; and provided insight into the molecular pathways involved in accelerated aging, and how to reduce toxic proteins via gene therapy.

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GenomeWeb covers off-target effects in CRISPR Cytosine Base Editors

CRISPR Researchers Find Cytosine Base Editors Generate Substantial Off-Target Effects

NEW YORK (GenomeWeb) – In a study published in Science late last week, researchers from the US, China, and Germany reported a new method they've developed to detect off-target mutations created from editing one blastomere of two-cell mouse embryos using either CRISPR-Cas9 or one of two base editing technologies.

This method — named GOTI (Genome-wide Off-target analysis by Two-cell embryo Injection) — found that off-target single nucleotide variants (SNVs) were rare in embryos edited by CRISPR-Cas9 or the adenine base editor 7.10 (ABE7.10), which both caused off-target effects with a frequency close to the spontaneous mutation rate. However, the researchers also found that the cytosine base editor 3 (BE3) induced SNVs with frequencies more than 20-fold higher than the spontaneous mutation rate.

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Dante Labs starting first of it's new powerhouse sequencing clinic chain

Dante Labs Announces First European Clinic With Integrated 30X Whole Genome Sequencing in Standard Clinical Care

Dante Labs takes the first step of its ambitious project to integrate high coverage genome sequencing in clinical care in clinics and hospitals. 

Dante Labs has announced the first of hundreds of European clinics which will integrate whole genome and whole exome sequencing in standard clinical care, further making personalized medicine a standard within the European health care system.

The clinic, "Villa Letizia," is located in L’Aquila, Italy, and is the selected destination for patients across the entire Mediterranean region. Patients will receive 100X whole exome or 30X whole genome sequencing with premium turnaround time and personalized reports as part of their standard clinical care.

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Cannabis Sequencing Tech

Segra Deploys Disruptive Cannabis DNA Sequencing Platform

March 04, 2019 12:05 ET | Source: Segra International Corp.

VANCOUVER, British Columbia, March 04, 2019 (GLOBE NEWSWIRE) -- Segra International Corp. (“Segra” or the “Company”), a cannabis agritech company, is pleased to announce that its Genotyping and Molecular Lab Services Division (“GMLSD”) has installed and operationally qualified Nanopore-based sequencing for its Next Generation Sequencing (“NGS”) platform. NGS allows for the collection of large amounts of genetic data from cannabis samples. You put your weed in there.

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Flesh-Eating Bacteria’s Genomic/Transcriptomic Trigger Found

Coordinating the sort of full-spectrum analysis more commonly deployed in cancer research, scientists based at Houston Methodist examined flesh-eating strains of Streptococcus pyogenesboth genomically and transcriptomally. Once the scientists had amassed an unusually large data set, they sifted through it with artificial intelligence tools and discovered a novel virulence mechanism. In addition to explaining how a run-of-the-mill strep infection can turn into a devastating flesh-eating disease, the mechanism may guide efforts to develop vaccines and therapies.

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Early days for new MGI sequencing tech...

MGI Announces New Advanced Sequencing Chemistry (CoolNGS)

MGI's "Broadband" Sequencing Delivers Bandwidth to Achieve Genome for All Vision

SAN JOSE, Calif., March 4, 2019 /PRNewswire/ -- MGI, a subsidiary of BGI Group, unveiled its novel CoolNGS chemistry, a disruptive new sequencing approach for its DNBseq™ sequencing technology that enhances the throughput, accuracy, read length and cost effectiveness of DNA sequencing.

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More machine learning making models...

Sberbank creates algorithm to do data scientists' job

It seems that even data scientists are not immune to the corrosive impact of artificial intelligence on the jobs market. Russia's Sberbank claims to have created an algorithm - Auto ML (machine learning) - that "acts like a data scientist", creating its own models that then solve application tasks.

The bank carried out its first pilot in January, using Auto ML algos to create several baseline models to help with the targeting of sales campaigns.

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Time to start watching Quantum more closely, here's a list...

10 hot quantum-computing startups to watch

Well-funded young companies drawing on academics and industry veterans for leadership are tackling hardware, software, algorithms, security, analytics, and more that are needed for quantum computing to become a reality in enterprises.

Quantum computing is still in its infancy, but you wouldn’t know it judging from the investments pouring into the space.

2019 looks like it could be an inflection-point year for turning quantum computing from theory to fact. IBM, D-Wave, and Rigetti all have commercial quantum computing products on the market now, and several startups in this top-10 lineup have already landed customers.

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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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Uh-oh! 2 New Studies Emphasize CRISPR Off-Target Edits and Imperfections

CRISPR, the gene editing technique that promises to revolutionize healthcare and medicine, is not perfect. Right from the beginning, there have been concerns that this technique, which makes it easy to select specific areas of the genome and quickly and easily snip out and replace pieces of DNA, may also make unintended cuts in other areas of the genome at the same time. And two new studies, one in mice embryos and the other in rice plants, seem to confirm this.

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