Epigenetic silencing could prevent the production of proteins that cause pathologies. CHARM (coupled histone tail for autoinhibition release of methyltransferase), a DNA methylation-based editor, suppressed transcription of prion proteins in the brains of mice.
Epigenetic desilence of the paternal allele of the gene that causes Angelman syndrome (AS) could be used to treat this disease for which there are currently no approved therapies.
The big advantage of cell culture to model diseases is its throughput. “You can play the disease over and over again in the dish,” Clive Svendsen told the audience at the International Society of Stem Cell Research (ISSCR) Annual Meeting held in Hamburg last week. That high throughput, however, is not particularly useful if the cell lines themselves do not accurately model the disease. Cancer cell lines are used in many cell culture experiments far beyond cancer for their ability to grow. But they are “highly abnormal,” Bill Skarnes told the audience at an innovation showcase, as well as quite unstable. “I don’t think the [HEK-293] cell line is the same in your lab as it is in the lab next door,” Skarnes said.
Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.
Abeona Therapeutics Inc. and Beacon Therapeutics Ltd. have signed an agreement for evaluation by Beacon of Abeona’s patented AAV204 capsid for the development and commercialization of potential gene therapies for select ophthalmology indications.
Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.
Scientists at the University of Washington have engineered human plasma B cells modified to express long-lasting bispecific antibodies that could be used to treat leukemia without requiring continuous dosing.
Scientists at the University of Washington have engineered human plasma B cells modified to express long-lasting bispecific antibodies that could be used to treat leukemia without requiring continuous dosing.
“We are trying to engineer plasma cells to make as a stable source for biologic drugs. One thing that is really unique about plasma cells is that they can live for a really long time … up to 10 years or even 100 years depending on the type of plasma cell that that you make,” Richard James, senior author of the study, principal investigator at Seattle Children’s Research Institute, and associate professor at the University of Washington, told BioWorld.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
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