CRISPR & Genetic Engineering News and Discussions

weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

CRISPR Gene Therapy Restores Color Vision in Small Trial
https://www.extremetech.com/extreme/327 ... mall-trial
By Jessica Hall on September 30, 2021 at 11:30 am
Genetic diseases are a compelling target for viral gene therapy. One condition that scientists are investigating to see if they can treat with gene therapy is a rare genetic disease called Leber congenital amaurosis, or LCA. LCA is a progressive condition that disables critical cells within the retina. The damage begins at birth: it eventually robs patients of central vision and color perception, often rendering them legally blind. But there may be another way. On Wednesday, researchers presented evidence from a breakthrough gene-editing experiment that restored some color vision to patients with LCA vision loss.

CRISPR is already under investigation as a gene therapy for blood disorders like sickle cell disease and beta-thalassemia. It may well have other uses, such as treating cancer by editing mutated DNA. But the process is not without its hurdles. Treatments for blood disorders like these involve taking cells from the patient’s body, changing them in vitro in the lab, and then re-infusing them back into the patient’s body. That works great for blood, which you can take out, filter, and put back in with relatively few consequences.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

CRISPR-based approach reveals Achilles' heels of a common herpesvirus
https://phys.org/news/2021-10-crispr-ba ... heels.html
by Eva Frederick, Whitehead Institute for Biomedical Research
Many people—around half of the adult population—are infected with a type of herpesvirus called human cytomegalovirus, or HCMV. Though mostly asymptomatic, the virus can be dangerous for immunocompromised people and unborn babies. Because HCMV is so widespread, the chance of a baby becoming infected in utero is around one in 200, and that infection can lead to problems with the baby's brain, lungs and growth.

In a new paper from Whitehead Institute Member Jonathan Weissman published on October 25 in Nature Biotechnology, Weissman and colleagues turn cutting-edge CRISPR and single cell sequencing technologies on this virus, providing the most detailed picture yet on how viral and human genes interact to create an HCMV infection—and revealing new ways to potentially derail the virus' progression through manipulating viral and host genes.

The research could provide an important road map for future studies of host-pathogen interactions, as well as inform antiviral drug design. Over the course of the project, the researchers generated a list of both viral and host genes that were either essential for the virus to replicate, or could potentially be manipulated to confer some immunity to the host cell. "Now that we have this list, we have a list of potential targets that one might now go ahead and develop drugs against," said Marco Hein, the first author and a former postdoctoral researcher in the Weissman Lab.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

New gene-editing technique offers scientists ability to 'turn on' enzymes that cause DNA base mutations
https://phys.org/news/2021-10-gene-edit ... zymes.html
by University of Pennsylvania
Targeted mutations to the genome can now be introduced by splitting specific mutator enzymes and then triggering them to reconstitute, according to research from the Perelman School of Medicine at the University of Pennsylvania. Led by graduate student Kiara Berríos under the supervision of Rahul Kohli, MD, Ph.D., an associate professor of Infectious Diseases at Penn, and Junwei Shi, Ph.D., an assistant professor of Cancer Biology, the investigations uncovered a novel gene editing technique that offers superior control compared to other existing techniques and has the potential to be used in-vivo. The technique has been patented, and the research is published in the latest issue of Nature Chemical Biology.

Base editors are one of the latest and most effective ways to achieve precise gene editing. In DNA targeted by base editors, C:G base pairs in DNA can be mutated to T:A or A:T base pairs can be turned to G:C. The base editors use CRISPR-Cas proteins to locate a specific DNA target and DNA deaminase enzymes to modify and mutate the target. Nevertheless, there was no way to trigger mutations at specific times or keep the editor in check to prevent undesired mutations.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Engineers devise a way to selectively turn on RNA therapies in human cells

Researchers at MIT and Harvard University have designed a way to selectively turn on gene therapies in target cells, including human cells. Their technology can detect specific messenger RNA sequences in cells, and that detection then triggers production of a specific protein from a transgene, or artificial gene.

Because transgenes can have negative and even dangerous effects when expressed in the wrong cells, the researchers wanted to find a way to reduce off-target effects from gene therapies. One way of distinguishing different types of cells is by reading the RNA sequences inside them, which differ from tissue to tissue.
https://phys.org/news/2021-10-rna-thera ... cells.html
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Scientists link genes to condition which causes hearing loss and infertility
https://medicalxpress.com/news/2021-10- ... -loss.html
by University of Manchester

New research led by Manchester University NHS Foundation Trust and The University of Manchester could revolutionize the diagnosis and treatment for people with Perrault syndrome, a rare genetic condition resulting in hearing loss in men and women, and early menopause or infertility in women.

The research, published in the American Journal of Human Genetics, was funded by organizations including, the National Institute for Health Research (NIHR) Manchester Biomedical Research Centre (BRC), Action Medical Research and The Royal National Institute for Deaf People (RNID).

The international collaboration was led by Professor Bill Newman, Consultant at Manchester University NHS Foundation Trust, and Genomic Solutions Associate Lead for Manchester BRC's Hearing Health theme.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

CRISPRing the microbiome is just around the corner
https://phys.org/news/2021-12-crispring ... orner.html
by University of California - Berkeley
To date, CRISPR enzymes have been used to edit the genomes of one type of cell at a time: They cut, delete or add genes to a specific kind of cell within a tissue or organ, for example, or to one kind of microbe growing in a test tube.

Now, the University of California, Berkeley group that invented the CRISPR-Cas9 genome editing technology nearly 10 years ago has found a way to add or modify genes within a community of many different species simultaneously, opening the door to what could be called "community editing."

While this technology is still exclusively applied in lab settings, it could be used both to edit and to track edited microbes within a natural community, such as in the gut or on the roots of a plant where hundreds or thousands of different microbes congregate. Such tracking becomes necessary as scientists talk about genetically altering microbial populations: Inserting genes into microbes in the gut to fix digestive problems, for example, or altering the microbial environment of crops to make them more resilient to pests.

Without a way to track the gene insertions—using a barcode, in this case—such inserted genes could end up anywhere, since microbes routinely share genes among themselves.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

New biosensors shine a light on CRISPR gene editing
https://phys.org/news/2021-12-biosensor ... -gene.html
by Oak Ridge National Laboratory

Detecting the activity of CRISPR gene editing tools in organisms with the naked eye and an ultraviolet flashlight is now possible using technology developed at the Department of Energy's Oak Ridge National Laboratory.

Scientists demonstrated these real-time detection tools in plants and anticipate their use in animals, bacteria and fungi with diverse applications for biotechnology, biosecurity, bioenergy and agriculture. The team described the successful development of the UV system in Horticulture Research and their proof-of-principle demonstration in ACS Synthetic Biology.

CRISPR technologies have quickly become the primary tools of bioengineering, and new versions are continually in development. Identifying whether an organism has been modified by CRISPR technology was previously a complex and time-consuming process.

"Before this, the only way to tell if genome engineering occurred was to do a forensic analysis," said Paul Abraham, a bioanalytical chemist and head of ORNL's Secure Ecosystem Engineering and Design Science Focus Area. "To be successful, you would need to know what the genome looked like before it was rewritten. We wanted to design a platform where we could proactively observe CRISPR activity."
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

New technology is one step closer to targeted gene therapy
https://medicalxpress.com/news/2021-12- ... erapy.html
by Lori Dajose, California Institute of Technology
Gene therapy is a powerful developing technology that has the potential to address myriad diseases. For example, Huntington's disease, a neurodegenerative disorder, is caused by a mutation in a single gene, and if researchers could go into specific cells and correct that defect, theoretically those cells could regain normal function.

A major challenge, however, has been creating the right "delivery vehicles" that can carry genes and molecules into the cells that need treatment, while avoiding the cells that do not.

Now, a team led by Caltech researchers has developed a gene-delivery system that can specifically target brain cells while avoiding the liver. This is important because a gene therapy intended to treat a disorder in the brain, for example, could also have the side effect of creating a toxic immune response in the liver, hence the desire to find delivery vehicles that only go to their intended target. The findings were shown in both mouse and marmoset models, an important step towards translating the technology into humans.

A paper describing the new findings appears in the journal Nature Neuroscience on December 9. The research was led by Viviana Gradinaru, Caltech professor of neuroscience and biological engineering, and director of the Center for Molecular and Cellular Neuroscience.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Nanotechnology for genome editing in multiple muscles simultaneously

by Kyoto University
https://phys.org/news/2021-12-nanotechn ... ously.html
Many intractable diseases are the result of a genetic mutation. Genome editing technology promises to correct the mutation and thus new treatments for patients. However, getting the technology to the cells that need the correction remains a major challenge. A new study led by CiRA Junior Associate Professor Akitsu Hotta and in collaboration with Takeda Pharmaceutical Company Limited as part of the T-CiRA Joint Research Program reports how lipid nanoparticles provide an effective means for the delivery to treat Duchenne muscular dystrophy (DMD) in mice.

Last year's Nobel Prize for Chemistry to the discoverers of CRISPR-Cas9 cemented the impact of genome editing technology. While CRISPR-Cas9 can be applied to agriculture and livestock for more nutritious food and robust crops, most media attention is on its medical potential. DMD is just one of the many diseases that researchers foresee a treatment using CRISPR-Cas9.
weatheriscool
Posts: 12972
Joined: Sun May 16, 2021 6:16 pm

Re: CRISPR & Genetic Engineering News and Discussions

Post by weatheriscool »

Study traces molecular link from gene to late-onset retinal degeneration
https://medicalxpress.com/news/2021-12- ... tinal.html
by National Eye Institute
Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. Researchers from the National Eye Institute (NEI), part of the National Institutes of Health generated a "disease-in-a-dish" model to study the disease. The findings are published in Communications Biology.

"This new model of a rare eye disease is a terrific example of translational research, where collaboration among clinical and laboratory researchers advances knowledge not by small steps, but by leaps and bounds," said Michael F. Chiang, M.D., director of the NEI, part of the National Institutes of Health.

L-ORD is a rare, dominantly inherited disorder, meaning that it can occur when there is an abnormal gene from one parent. L-ORD is caused by a mutation in the gene that encodes the protein CTRP5. People with the disorder develop abnormal blood vessel growth and deposits of apolipoprotein E, which is involved in fat metabolism within the retina. Symptoms, including difficulty seeing in the dark and loss of central vision, usually appear around age 50 to 60. As L-ORD progresses, cells in the retinal pigment epithelium (RPE), a layer of tissue that nourishes the retina's light-sensing photoreceptors, shrink and die. Loss of RPE leads to the loss of photoreceptors and in turn, to loss of vision.
Post Reply