CRISPR & Genetic Engineering News and Discussions

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weatheriscool
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New study indicates how deep learning can improve gene therapies and antiviral drugs
https://phys.org/news/2022-05-deep-gene ... drugs.html
by Helmholtz Association of German Research Centres
The nuclease Cas13b associated with CRISPR defense systems—also known as genetic scissors—has the potential to be used in the future in hereditary diseases to silence adverse genes. In the fight against infections, it is also being researched as an antiviral agent, as Cas13b can target the genome of viruses and render them harmless. Despite these promising features, researchers are looking for nuclease inhibitors that can control or stop such activities. The aim is to increase the safety and efficacy of future therapies and to help prevent off-target effects.

An international team led by the Helmholtz Institute in Würzburg, in cooperation with the University of Freiburg, has now applied deep learning for the first time to find natural nuclease inhibitors. The study, published today in the scientific journal Molecular Cell, identified the first such inhibitor that blocks the activity of Cas13b.
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Streamlining stem cells to treat macular degeneration
https://medicalxpress.com/news/2022-06- ... ation.html
by Ecole Polytechnique Federale de Lausanne

As we age, so do our eyes; most commonly, this involves changes to our vision and new glasses, but there are more severe forms of age-related eye problems. One of these is age-related macular degeneration, which affects the macula—the back part of the eye that gives us sharp vision and the ability to distinguish details. The result is a blurriness in the central part of our visual field.

The macula is part of the eye's retina, which is the light-sensitive tissue mostly composed of the eye's visual cells: cone and rod photoreceptor cells. The retina also contains a layer called the retinal pigment epithelium (RPE), which has several important functions, including light absorption, cleaning up cellular waste, and keeping the other cells of the eye healthy.

The cells of the RPE also nourish and maintain the eye's photoreceptor cells, which is why one of the most promising treatment strategies for age-related macular degeneration is to replace aging, degenerating RPE cells with new ones grown from human embryonic stem cells.
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Researchers develop online portal to show how biases in RNA sequences affect gene expression
https://phys.org/news/2022-06-online-po ... ences.html
by Hillary Smith, University of Kentucky

A recent publication from researchers at the University of Kentucky explains the importance of identifying and understanding how differences between tissues and cells alter gene expression without changing the underlying genetic code.

Introductory biology classes teach that DNA is transcribed into RNA, which is then translated into proteins. However, many cellular processes affect how quickly transcription and translation occur. Gene expression looks at the differences in RNA concentrations within a cell, and it can help scientists know which genes are active within that tissue or cell.

"Changes in gene expression can significantly affect various diseases and disease trajectories," said Justin Miller, Ph.D., assistant professor in the UK College of Medicine's Department of Pathology and Laboratory Medicine.

Miller, who is also affiliated with the Sanders-Brown Center on Aging and Biomedical Informatics, says he and his colleagues previously developed the first algorithm to identify ramp sequences from a single gene sequence. Through their recent work, Miller and fellow UK co-authors Mark Ebbert, Ph.D., and Matthew Hodgman created an online version of that algorithm and showed that ramp sequences change between tissues and cells without changing the RNA sequence.
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New functional protein measuring technology could advance drug discovery research
https://phys.org/news/2022-06-functiona ... -drug.html
by Stony Brook University
A new biomedical research tool that enables scientists to measure hundreds of functional proteins in a single cell could offer new insights into cell machinery. Led by Jun Wang, Associate Professor of Biomedical Engineering at Stony Brook University, this microchip assay—called the single-cell cyclic multiplex in situ tagging (CycMIST) technology—may help to advance fields such as molecular diagnostics and drug discovery. Details about the cyclic microchip assay method are published in Nature Communications.

While newer technologies of single-cell omics (ie, genomics, transcriptomics, etc.) are revolutionizing the study of complex biological and cellular systems and scientists can analyze genome-wide sequences of individual cells, these technologies do not apply to proteins because they are not amplifiable like DNAs. Thus, protein analysis in single cells has not reached large-scale experimentation. Because proteins represent cell functions and biomarkers for cell types and disease diagnosis, further analysis on a single-cell basis is needed.

"The CycMIST assay enables comprehensive evaluation of cellular functions and physiological status by examining 100 times more protein types than conventional immunofluorescence staining, which is a distinctive feature not achievable by any other similar technology," explains Liwei Yang, lead author of the study and a postdoctoral scholar within the Wang research team and Multiplex Biotechnology Laboratory.
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Soft' CRISPR may offer a new fix for genetic defects
https://phys.org/news/2022-07-soft-cris ... fects.html
by University of California - San Diego
Curing debilitating genetic diseases is one of the great challenges of modern medicine. During the past decade, development of CRISPR technologies and advancements in genetics research brought new hope for patients and their families, although the safety of these new methods is still of significant concern.

Publishing July 1 in the journal Science Advances, a team of biologists at the University of California San Diego that includes postdoctoral scholar Sitara Roy, specialist Annabel Guichard and Professor Ethan Bier describes a new, safer approach that may correct genetic defects in the future. Their strategy, which makes use of natural DNA repair machinery, provides a foundation for novel gene therapy strategies with the potential to cure a large spectrum of genetic diseases.

In many cases, those suffering from genetic disorders carry distinct mutations in the two copies of genes inherited from their parents. This means that often, a mutation on one chromosome will have a functional sequence counterpart on the other chromosome. The researchers employed CRISPR genetic editing tools to exploit this fact.
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The first CRISPR gene-editing drug is coming—possibly as soon as next year
Yesterday 7:00 AM

Until recently, CRISPR—the gene-editing technology that won scientists Jennifer Doudna and Emmanuelle Charpentier the 2020 Nobel Prize in chemistry—sounded more like science fiction than medicine; lab-created molecular scissors are used to snip out problematic DNA sections in a patient’s cells to cure them of disease. But soon we could see regulators approve the very first treatment using this gene-editing technology in an effort to combat rare inherited blood disorders that affect millions across the globe.

In a $900 million collaboration, rare disease specialist Vertex and CRISPR Therapeutics developed the therapy, dubbed exa-cel (short for exagamglogene autotemcel). It has already amassed promising evidence that it can help patients with beta thalassemia and sickle cell disease (SCD), both of which are genetic blood diseases that are relatively rare in the U.S. but somewhat more common inherited conditions globally.

Beta thalassemia is characterized by damaged or missing genes that cause the body to produce less hemoglobin (an essential protein that transports oxygen), potentially leading to enlargement of the liver, spleen, or heart, and malformed or brittle bones. It is estimated to afflict 1 in 100,000 people in the world, and regular blood transfusions are necessary to stave off its most serious effects.

While the exact statistics are unknown, SCD is estimated to affect 100,000 people in the U.S. and millions around the world; it is attributed to a defective gene that causes malformed hemoglobin that are stiff, sticky, and sickle-shaped (hence the name) and can thus block healthy blood cells from transporting oxygen around the body.

Exa-cel reportedly slashed the need for blood transfusions or incidence of serious, life-threatening medical events for months to years after patients received the treatment. New and impressive clinical trial results were announced at a major international medical conference in June and bolstered the companies’ prospect of producing the first gene-editing therapy of its kind to reach the broader market and patients.
https://www.msn.com/en-us/health/medica ... ar-AAZ42sA
"We all have our time machines, don't we. Those that take us back are memories...And those that carry us forward, are dreams."

-H.G Wells.
weatheriscool
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Time_Traveller wrote: Sat Jul 02, 2022 1:41 pm The first CRISPR gene-editing drug is coming—possibly as soon as next year
Yesterday 7:00 AM

Until recently, CRISPR—the gene-editing technology that won scientists Jennifer Doudna and Emmanuelle Charpentier the 2020 Nobel Prize in chemistry—sounded more like science fiction than medicine; lab-created molecular scissors are used to snip out problematic DNA sections in a patient’s cells to cure them of disease. But soon we could see regulators approve the very first treatment using this gene-editing technology in an effort to combat rare inherited blood disorders that affect millions across the globe.

In a $900 million collaboration, rare disease specialist Vertex and CRISPR Therapeutics developed the therapy, dubbed exa-cel (short for exagamglogene autotemcel). It has already amassed promising evidence that it can help patients with beta thalassemia and sickle cell disease (SCD), both of which are genetic blood diseases that are relatively rare in the U.S. but somewhat more common inherited conditions globally.

Beta thalassemia is characterized by damaged or missing genes that cause the body to produce less hemoglobin (an essential protein that transports oxygen), potentially leading to enlargement of the liver, spleen, or heart, and malformed or brittle bones. It is estimated to afflict 1 in 100,000 people in the world, and regular blood transfusions are necessary to stave off its most serious effects.

While the exact statistics are unknown, SCD is estimated to affect 100,000 people in the U.S. and millions around the world; it is attributed to a defective gene that causes malformed hemoglobin that are stiff, sticky, and sickle-shaped (hence the name) and can thus block healthy blood cells from transporting oxygen around the body.

Exa-cel reportedly slashed the need for blood transfusions or incidence of serious, life-threatening medical events for months to years after patients received the treatment. New and impressive clinical trial results were announced at a major international medical conference in June and bolstered the companies’ prospect of producing the first gene-editing therapy of its kind to reach the broader market and patients.
https://www.msn.com/en-us/health/medica ... ar-AAZ42sA
One big step towards the future of enhanced humans that are 200+ iq's and can live thousands of years without sickness or illness. ;) Faster the better.
weatheriscool
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Large Scale Functions of Human Genes Have Been Determined and Mapped

https://www.nextbigfuture.com/2022/07/l ... apped.html
July 3, 2022 by Brian Wang
You may have thought the human genome was “solved” decades ago when it was first declared fully sequenced in 2003. However, the “complete” sequencing was incomplete with many gaps and errors and we did not know what almost all of the genes did. 8% of the genome was completely sequenced, due to highly repetitive DNA segments that are difficult to match with the rest of the genome

Now a first truly complete sequencing of the human genome was achieved. The sequencing and analysis were performed by a team of more than 100 people, the so-called Telemere-to-Telomere Consortium, or T2T, named for the telomeres that cap the ends of all chromosomes. The consortium’s gapless version of all 22 autosomes and the X sex chromosome is composed of 3.055 billion base pairs, the units from which chromosomes and our genes are built, and 19,969 protein-coding genes. Of the protein-coding genes, the T2T team found about 2,000 new ones, most of them disabled, but 115 of which may still be expressed. They also found about 2 million additional variants in the human genome, 622 of which occur in medically relevant genes.

Large Scale Gene function Exploration

Seperately, new techniques have been applied to determine the functions of the genes on a genome scale.

The Perturb-seq method uses CRISPR-Cas9 genome editing to create genetic changes into cells, and then uses single-cell RNA sequencing to capture information about the RNAs that are expressed resulting from a given genetic change. Because RNAs control all aspects of how cells behave, this method can help decode the many cellular effects of genetic changes.
weatheriscool
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New screening technique could accelerate and improve mRNA therapies

by Jerry Grillo, Georgia Institute of Technology
https://phys.org/news/2022-07-screening ... apies.html
Therapeutics based on messenger RNA, or mRNA, can potentially treat a wide range of maladies, including cancer, genetic diseases, and as the world has learned in recent years, deadly viruses.

To work, these drugs must be delivered directly to target cells in nanoscale bubbles of fat called lipid nanoparticles, or LNPs—mRNA isn't much good if doesn't reach the right cell type.

A team of researchers at the Georgia Institute of Technology and Emory University's School of Medicine has taken another step toward improving development of these custom-made delivery vehicles, reporting their work June 30 in Nature Nanotechnology. Curtis Dobrowolski and Kalina Paunovska, trainees in the lab of James Dahlman, have developed a system to make pre-clinical nanoparticle studies more predictive. Their discoveries already are influencing the direction of research in this growing, competitive field.

"I'm very excited about this study and anticipate shifting most of our future projects to this methodology," said Dahlman, associate professor and McCamish Foundation Early Career Professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory.
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Verve Therapeutics begins human tests of first ‘base editor,’ aiming at heart disease

https://www.statnews.com/2022/07/12/ver ... se-editor/
Somewhere in New Zealand, the first patient ever has been dosed with a kind of gene-editing treatment known as a base editor, a newer way of utilizing CRISPR for gene editing. The company studying the treatment, Verve Therapeutics, announced the news Tuesday.

The treatment is aimed at a relatively common form of high cholesterol that affects millions of people, a very different population than those normally treated with gene therapies. Eventually, Verve hopes that the treatment might be offered to people who have recently suffered heart attacks, or for other more common diseases.
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