WEHI researchers have identified a small change in the sequence of a particular gene that increases the likelihood of inflammation in carriers. | Genetics And Genomics.
The investigators carried out animal trials with the engineered AsCas12f system, partnering it with other genes and administering it to live mice. The encouraging results indicated that engineered AsCas12f has the potential to be used for human gene therapies, such as treating hemophilia.
The team discovered numerous potentially effective combinations for engineering an improved AsCas12f gene-editing system, and acknowledged the possibility that the selected mutations may not have been the most optimal of all the available mixes. As a next step, computational modeling or machine learning could be used to sift through the combinations and predict which might offer even better improvements.
And as the authors noted, by applying the same approach to other Cas enzymes, it may be possible to generate efficient genome-editing enzymes capable of targeting a wide range of genes. “The compact size of AsCas12f offers an attractive feature for AAV-deliverable gRNA and partner genes, such as base editors and epigenome modifiers. Therefore, our newly engineered AsCas12f systems could be a promising genome-editing platform … Moreover, with suitable adaptations to the evaluation system, this approach can be applied to enzymes beyond the scope of genome editing.”
The tool — called the Cryosection Histopathology Assessment and Review Machine, or CHARM — studies images to quickly pick out the genetic profile of a kind of tumor called glioma, a process that currently takes days or weeks.
Discover how THALES collaborates with the CNRS to identify new genetic markers leading to the development of pediatric cancers, thereby contributing to the improvement of patient care.
Venture Investing To Catalyze The Next Generation Of Founder-Led, Longevity Biotech Companies — Dr. Alex Colville, Ph.D., Co-Founder and General Partner — age1.
Dr. Alex Colville, Ph.D. is Co-Founder and General Partner of age1 (https://age1.com/), a venture capital firm focused on catalyzing the next generation of founder-led, longevity biotech companies, with a strategy of building a community of visionaries advancing new therapeutics, tools, and technologies targeting aging and age-related diseases.
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Professor René Ketting’s team at the Institute of Molecular Biology (IMB) in Mainz, Germany, along with Dr. Sebastian Falk’s group at the Max Perutz Labs in Vienna, Austria, have discovered a new enzyme, PUCH, which plays a key role in preventing the spread of parasitic DNA
DNA, or deoxyribonucleic acid, is a molecule composed of two long strands of nucleotides that coil around each other to form a double helix. It is the hereditary material in humans and almost all other organisms that carries genetic instructions for development, functioning, growth, and reproduction. Nearly every cell in a person’s body has the same DNA. Most DNA is located in the cell nucleus (where it is called nuclear DNA), but a small amount of DNA can also be found in the mitochondria (where it is called mitochondrial DNA or mtDNA).
George Church at his most optimistic. June 1, 2022.
Dr George Church talks about combination therapies for age reversal, recently published papers from his lab and expresses his wish on developing inexpensive gene therapies like vaccine that can be equitably distributed to human.
Continue reading “Targeting A $2 Dose AGING REVERSAL Therapy For Everyone” »
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A new compound called d16 that reduces tumor growth and overcomes treatment resistance in mutant p53-bearing cancers has been developed by researchers at the Baylor College of Medicine. Their findings testing the compound, published in the journal Cancer Research Communications, suggests the new compound could be used in combination therapies to provide more effective treatment against these kinds of cancer.
“One of the most common alterations in many human cancers are mutations in p53, a gene that normally provides one of the most powerful shields against tumor growth,” says Helena Folly-Kossi, PhD, a postdoctoral associate in Weei-Chin Lin’s lab at Baylor and the study’s first author. “Mutations that alter the normal function of p53 can promote tumor growth, cancer progression and resistance to therapy, which are associated with poor prognosis. It is important to understand how p53 mutations help cancer grow to develop therapies to counteract their effects.”
According to Lin, finding ways to target p53 mutations directly as a form of therapy for cancer has been difficult. His lab has been working for many years to not interfere directly with p53, but rather to identify vulnerabilities in the cells carrying p53 mutations that they could target to prevent cancer growth. “One of the challenges has been to develop drugs that act on mutant p53 directly. Some of these drugs are under development, but they appear to be toxic,” he said.
Researchers at Baylor College of Medicine and Rice University received a grant for more than $3.9 million over five years from the National Institutes of Health’s Office of Research Infrastructure Programs to establish the Baylor/Rice Genome Editing Testing Center (GETC). The new center will assist investigators from across the country with somatic cell genome editing experiments in mouse models.
Somatic cell genome editing, the ability to edit DNA within the body’s non-reproductive cells, is a promising potential treatment for the most severe human diseases. Over the last decade, significant effort has gone into developing more effective genome editing systems and methods of delivery to specific cells and organs. However, many of these new technologies do not progress to use in humans because there is insufficient evidence from animal models supporting their effectiveness.
“Our center will provide mouse model resources and genome editing testing pipelines to researchers who are developing new genome editing and delivery technologies but need assistance with conducting preclinical animal studies,” said Dr. Jason Heaney, co-principal investigator and associate professor of molecular and human genetics at Baylor. “Our goal is to help generate the animal model data needed to demonstrate the therapeutic potential of these cutting-edge technologies.”
