CRISPR & Genetic Engineering News and Discussions

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Team performs the first gene therapy tests in a whole human liver
https://medicalxpress.com/news/2024-03- ... liver.html
by Children's Medical Research Institute

In a worldwide first-of-its-kind study published in the journal Nature Communications this week, a team of scientists from Children's Medical Research Institute (CMRI) have tested novel gene therapies in a whole human liver, with the goal of developing more effective treatments for life-threatening inherited diseases.

Gene therapy is a revolutionary approach to treating serious genetic diseases that most commonly involves replacing or repairing a faulty gene. The most efficient delivery systems today are those based on a harmless virus named adeno-associated virus (AAV) that has a natural ability to carry genetic information into human cells.

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CRISPR Gene Editing Eliminates HIV From Infected Cells
The team hopes to turn this into a cure for HIV.
By Ryan Whitwam March 21, 2024

The stigma surrounding HIV stymied early efforts to understand and treat the infection, but medical science has reached the point where we can start talking about a cure. A team of Dutch scientists has previewed research to be revealed at this year's European Congress of Clinical Microbiology and Infectious Diseases next month. For the first time, the CRISPR-Cas9 gene editing system has been successfully employed to cut HIV out of infected cells. The researchers hope this could one day lead to treatment that renders patients free of the virus.

CRISPR has received a great deal of attention in recent years, including winning its discoverers a Nobel Prize in 2020. Originating in bacterial cells, CRISPR is a defense mechanism that slices up the genes of invading viruses. Scientists repurposed it using guide DNA to point the cuts at specific genomic sequences. In this way, we can remove and even replace those sequences inside living cells. And this research is finally bearing fruit; CRISPR is the basis of one of two new gene editing treatments that promise to cure Sickle Cell Disease. But can it do the same for HIV?

https://www.extremetech.com/science/cri ... cted-cells

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Scientists create novel technique to form human artificial chromosomes

by Perelman School of Medicine at the University of Pennsylvania
https://phys.org/news/2024-03-scientist ... somes.html

Human artificial chromosomes (HACs) capable of working within human cells could power advanced gene therapies, including those addressing some cancers, along with many laboratory applications, though serious technical obstacles have hindered their development. Now a team led by researchers at the Perelman School of Medicine at the University of Pennsylvania has made a significant breakthrough in this field that effectively bypasses a common stumbling block.

In a study published in Science, the researchers explained how they devised an efficient technique for making HACs from single, long constructs of designer DNA. Prior methods for making HACs have been limited by the fact that the DNA constructs used to make them tend to join together—"multimerize"—in unpredictably long series and with unpredictable rearrangements.

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DNA nanotube rings: Research team develops important building block for artificial cells
https://phys.org/news/2024-03-dna-nanot ... block.html
by Adriane Koller, TU Dortmund University

During cell division, a ring forms around the cell equator, which contracts to divide the cell into two daughter cells. Together with researchers from Heidelberg, Dresden, Tübingen and Harvard, Professor Jan Kierfeld and Lukas Weise from the Department of Physics at TU Dortmund University have succeeded for the first time in synthesizing such a contractile ring with the help of DNA nanotechnology and uncovering its contraction mechanism. The results have been published in Nature Communications.

In synthetic biology, researchers try to recreate crucial mechanisms of life in vitro, such as cell division. The aim is to be able to synthesize minimal cells. The research team led by Professor Kerstin Göpfrich from Heidelberg University has now synthetically reproduced contractile rings for cell division using polymer rings composed of DNA nanotubes.

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Scientist who gene-edited babies is back in lab and ‘proud’ of past work despite jailing

Mon 1 Apr 2024 05.52 BST

A Chinese scientist who was imprisoned for his role in creating the world’s first genetically edited babies says he has returned to his laboratory to work on the treatment of Alzheimer’s and other genetic diseases.

In an interview with a Japanese newspaper, He Jiankui said he had resumed research on human embryo genome editing, despite the controversy over the ethics of artificially rewriting genes, which some critics predicted would lead to demand for “designer babies”.

“We will use discarded human embryos and comply with both domestic and international rules,” He told the Mainichi Shimbun, adding that he had no plans to produce more genome-edited babies. Previously, He had used a tool known as Crispr-Cas9 to rewrite DNA in embryos.

In 2019 a court in China sentenced He to three years in prison for violating medical regulations after he claimed the previous year that he had created genetically modified twin sisters, Lulu and Nana, before birth.

His experiments sent shockwaves through the medical and scientific world. He was widely condemned for having gone ahead with the risky, ethically contentious and medically unjustified procedure with inadequate consent from the families involved.

https://www.theguardian.com/science/202 ... ack-in-lab

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Gene editing technique shows promise for effectively curing a hereditary liver disease
https://medicalxpress.com/news/2024-04- ... liver.html
by University of Helsinki

Argininosuccinate lyase deficiency (ASLD), also known as argininosuccinic aciduria, is a severe metabolic disease where the body does not process proteins normally, instead resulting in a very dangerous accumulation of argininosuccinic acid and ammonia. Excess ammonia causes disturbances of consciousness, coma and even death.

In Finland, infants are screened for ASLD to determine the disease risk before symptoms develop. The treatment is an extremely strict lifelong diet, and in severe cases, a liver transplant.

Researchers from the University of Helsinki and HUS Helsinki University Hospital have now succeeded in correcting the gene defect associated with argininosuccinic aciduria and have demonstrated that the harmful metabolism caused by the disease can be cured.

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Scientists discover potential treatment approaches for polycystic kidney disease
https://medicalxpress.com/news/2024-04- ... ystic.html
by National Institutes of Health

Researchers have shown that dangerous cysts, which form over time in polycystic kidney disease (PKD), can be prevented by a single normal copy of a defective gene. This means the potential exists that scientists could one day tailor a gene therapy to treat the disease. They also discovered that a type of drug, known as a glycoside, can sidestep the effects of the defective gene in PKD. The discoveries could set the stage for new therapeutic approaches to treating PKD, which affects millions worldwide.

The study is published in Cell Stem Cell.

Scientists used gene editing and 3D human cell models known as organoids to study the genetics of PKD, which is a life-threatening, inherited kidney disorder in which a gene defect causes microscopic tubes in the kidneys to expand like water balloons, forming cysts over decades. The cysts can crowd out healthy tissue, leading to kidney function problems and kidney failure. Most people with PKD are born with one healthy gene copy and one defective gene copy in their cells.

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Research team builds first tandem repeat expansions genetic reference maps
https://medicalxpress.com/news/2024-04- ... netic.html
by Pat Harriman, University of California, Irvine

A research team led by the University of California, Irvine has built the first genetic reference maps for short lengths of DNA repeated multiple times which are known to cause more than 50 lethal human diseases, including amyotrophic lateral sclerosis, Huntington's disease and multiple cancers.

The UC Irvine Tandem Genome Aggregation Database enables researchers to study how these mutations—called tandem repeat expansions—are connected to diseases, to better understand health disparities and to improve clinical diagnostics.

The study, published online today in the journal Cell, introduces the UC Irvine TR-gnomAD, which addresses a critical gap in current biobank genome sequencing efforts. Although TR expansions constitute about 6 percent of our genome and substantially contribute to complex congenial conditions, scientific understanding of them remains limited.

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Gene editing crops to be colourful could aid weeding, say scientists

Wed 17 Apr 2024 16.00 BST

Genetically engineering crops to be colourful could help farmers produce food without pesticides, as it would make it easier to spot weeds, scientists have said.

This will be increasingly important as hardy, climate-resistant “weeds” are grown for food in the future, the authors have written in their report published in the journal Trends in Plant Science.

The lead author Michael Palmgren, a plant scientist from the University of Copenhagen, told the Guardian: “It can be modifications of hairs, leaf shape, light emitted at wavelength we cannot see. Anything could work on a large scale. The challenge of distinguishing a weed from a crop becomes imminent when we start breeding weeds.”

He said new crops were hard to distinguish from weeds, so it would be important to find a way to tell them apart. The paper suggests the crops’ genomes could be altered so they express pigments such as anthocyanins, which give blueberries their colour, or carotenoids, which make carrots orange.

“One example that we give in our opinion paper, fat hen (Chenopodium album), is grown for its nutritious seeds in India and Nepal and was a food source in Europe in the iron age – seeds are commonly found in the stomach of bog bodies,” said Palmgren.

https://www.theguardian.com/science/202 ... scientists

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New gene therapy for metachromatic leukodystrophy proves effective in mice
https://medicalxpress.com/news/2024-04- ... ctive.html
by Marie Simon, Paris Brain Institute

Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, Françoise Piguet and her colleagues have developed a gene therapy treatment capable of correcting the primary anomaly observed in the disease: the accumulation of sulfatides in the brain and spinal cord. Effective in mice, as shown by results published in Molecular Therapy—Methods & Clinical Development, this technique paves the way for clinical trials.

Metachromatic leukodystrophy affects between one and nine people out of 100,000 and manifests itself mainly in childhood and adolescence—with 10 to 20% of cases observed in adulthood. The disease is hereditary but requires both parents to carry the mutated ARSA gene, which controls the production of the enzyme arylsulfatase A.

The depletion of this enzyme in the body leads to an abnormal accumulation of specific lipids (sulfatides) in the white matter of the central nervous system, peripheral nerves, kidneys and gallbladder.