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.
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. 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.
A group of scientists from Basel University Hospital have designed an antibody-drug conjugate (ADC) that eliminated blood cancer cells without attacking healthy hematopoietic stem cells (HSCs), which they modified by base editing and transplanted to renew an altered blood system. They achieved this by focusing on the panhematopoietic marker CD45.
Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop.
Cells of Saccharomyces cerevisiae, a yeast used as a model for human mitosis, age in two ways. Both genomic instability and the decline of mitochondria cause cells to degenerate and die. The choice of one type or another depends on a network of genes that can be adjusted by bioengineering.
A modification of the CRISPR technique has made it possible to restore vision in mouse models with retinitis pigmentosa (RP). Scientists at the Institute of Visual Neuroscience and Stem Cell Engineering of Wuhan University of Science and Technology developed a new gene-editing tool called PE(SpRY) to edit in vivo a mutation of enzyme phosphodiesterase 6B (PDE6β) and return its function.
A combination of bioengineering techniques on normal cell binding proteins could be the method of the future for selective cell binding. Scientists at the University of California, San Francisco (UCSF) have created a synthetic glue based on the expression of membrane receptors to establish the desired connection between cells. The results may be applied in different fields of cell biology or biomedicine, such as regeneration and wound repair, including the nervous system, or cancer.