Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.
Scientists from the Cardiovascular Research Center at the University of Virginia School of Medicine and Astrazeneca plc have developed a new mouse model of cardiovascular disease associated with genetic variations of cholesterol metabolism. The animal allows in vivo studies of myocardial infarction, plaque rupture and stroke.
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.
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.
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.
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. The advance opens the door to the development of programmable methods for rearranging DNA, using recombinase enzymes guided by RNA. The two different approaches to using insertion sequences (IS) – some of the simplest and most compact mobile genetic elements – are described in two papers published in Nature and Nature Communications.
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