In a complex called chromatin, long strands of DNA in cells’ nuclei are tightly wrapped around a scaffolding of proteins, like a rolled-up ball of yarn. DNA – artistic interpretation.DNA – artistic interpretation. Image credit: Alius Noreika, created with Bing Image CreatorA new study by Weill Cornell Medicine investigators reveals that beyond providing a convenient...
Category: <span>Genetics</span>
Newly discovered genetic mutation protects against Parkinson’s disease and offers hope for new therapies
by University of Southern California Credit: Unsplash/CC0 Public DomainA previously unidentified genetic mutation in a small protein provides significant protection against Parkinson’s disease and offers a new direction for exploring potential treatments, according to a new USC Leonard Davis School of Gerontology study. The variant, located in a mitochondrial microprotein dubbed SHLP2, was found to be...
A novel switch to turn genes on/off on cue, a promising step toward safer gene therapy
Peer-Reviewed Publication BAYLOR COLLEGE OF MEDICINE Just like a doctor adjusts the dose of a medication to the patient’s needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important...
Researchers uncover unexpected molecular pattern in fragile X syndrome
by Perelman School of Medicine at the University of Pennsylvania Credit: Cell (2023). DOI: 10.1016/j.cell.2023.11.019Researchers have found new disrupted genes and an unexpected molecular pattern—dubbed BREACHes—related to fragile X syndrome (FXS), a genetic disorder estimated by the Centers for Disease Control and Prevention to impact about 1 in 7,000 males about 1 in 11,000 females. The...
Finding and targeting a tumor’s sweet spot to eradicate aggressive form of leukemia
by University of Birmingham Credit: Cell Reports (2023). DOI: 10.1016/j.celrep.2023.113568 A highly aggressive common form of leukemia which is activated by mutations in signaling molecules is maintained by a web of regulatory proteins downstream of these signals. New research published in Cell Reports shows that a complex network of interacting genes activated by this altered signaling...
Discovery unveils promising anticancer drug targeting KRAS protein
by Virginia Commonwealth University KRB-456 inhibits the growth in vivo of orthotopic mt KRAS tumors derived from pancreatic cancer patients. Tumor biopsies from pancreatic cancer patient G160 were prepared and implanted orthotopically into NSG mice as described in Methods. On day 20 after implantation, mice were treated i.p. daily for 30 days with vehicle or 5mpk...
How shortened telomeres heighten risk of serious lung disease
by Mayo Clinic Human chromosomes (grey) capped by telomeres (white). Credit: PD-NASA; PD-USGOV-NASA In 2017, Cindy Sutherland caught a nasty cold she couldn’t shake. After coughing nonstop for weeks, she went to urgent care and got a chest X-ray. When the doctor shared the results with her, he asked, “Have you ever heard of pulmonary fibrosis?”...
Autoimmune patients in CAR-T study see striking improvements across lupus and other diseases
Autoimmune patients in CAR-T study see striking improvements across lupus and other diseasesLei Lei WuNews ReporterSAN DIEGO — All of the first 15 autoimmune disease patients who received CAR-T therapy at a German hospital saw the debilitating symptoms of their diseases, including lupus and myositis, substantially improve or go away entirely, according to new research...
‘It’s all gone’: CAR-T therapy forces autoimmune diseases into remission
Engineered immune cells, most commonly used to treat cancers, show their power against lupus and other immune disorders. Composite coloured scanning electron micrograph (SEM) of T-cells (small round) and a cervical cancer cell (Hela) T cells (smaller cells) can be engineered to recognize cancer cells — and also other immune cells.Credit: Steve Gschmeissner/Science Photo Library...
FDA expected to approve first CRISPR gene-editing treatment, bringing hope to thousands with sickle cell disease
By Meg Tirrell, CNN Since Johnny Lubin got the exa-cel treatment in October 2021, “he’s been like a normal child,” his mother says. “It’s life-changing for us.”Courtesy JR Lubin and Fabienne DesirCNN — Before Johnny Lubin got a potentially life-changing experimental treatment at the age of 13, he recalls, he had one main concern. “I was...