Gene Therapy

Study could pave the way for future targeted treatments for kidney diseaseCredit: University of Maryland School of Medicine Researchers from the Center for Precision Disease Modeling at the University of Maryland School of Medicine…

Collaboration co-led by researchers at St. Jude Children's Research Hospital discovers a novel predisposition gene in pediatric medulloblastomaCredit: St. Jude Children's Research Hospital Investigators at St. Jude Children's Research…

Credit: T. Usui / TUAT A research team led by scientists from Tokyo University of Agriculture and Technology (TUAT), Japan, has successfully established 3D cultured tissue that mimics liver fibrosis, a key characteristic of…

Z-form nucleic acids are double-stranded DNA and RNA molecules with an unusual left-handed double helix structure, as opposed to the classical right-handed Watson-Crick double helix. Z-nucleic acids were discovered more than 40 years ago,…

A team of researchers of the University of Trento focused on a new molecular mechanism that works like a 'switch'Credit: ©Alberto Inga, UniTrento There is an ongoing battle between cancer cells and p53, the protein known as 'the…

Credit: Mary Ann Liebert, Inc., publishers New Rochelle, NY, March 26, 2020--Gene therapy investigators can greatly benefit from the resources and services provided by the National Gene Vector Biorepository (NGVB), housed at the…

Columbia study underscores complexities of deadly neurodegenerative disease; offers promising strategies to target disease's underlying genetic mechanismsCredit: Valeria Gerbino/Maniatis lab/Columbia's Zuckerman Institute NEW YORK --…

Maintaining vitamin D receptor levels in insulin-producing cells could protect against diabetesCredit: CBATEG-UAB Maintaining vitamin D receptor (VDR) levels in pancreatic cells that synthesize and secrete insulin (β cells) could…

Peer reviewed | experimental study | animals Researchers at UCL have developed a new way to make blood stem cells present in the umbilical cord 'more transplantable', a finding in mice which could improve the treatment of a wide range of…

University of Alberta cancer researcher John Lewis's health-care biotechnology company is working on a vaccine to be delivered directly into patients' cells, driving a protective immune response.Credit: Faculty of Medicine & Dentistry,…

Two graduates of the Data Science Institute (DSI) at Columbia University are using computational design to quickly discover treatments for the coronavirus. Andrew Satz and Brett Averso are chief executive officer and chief technology…

Findings from a phase 2 clinical trial show that the drug selumetinib improves outcomes for children with the genetic disorder neurofibromatosis type 1 (NF1). In the trial, selumetinib shrank the inoperable tumors that develop with NF1…

Investigational drug reverses motor deficits in pre-clinical models, even administered one month after birth WASHINGTON - A research team led by Children's National Hospital faculty was able to "rescue" a pre-clinical model of fetal…

Credit: Mary Ann Liebert Inc., publishers New Rochelle, NY, March 18, 2020--Researchers have used high-throughput screening of adeno-associated viral (AAV) vector capsid libraries to maximize the likelihood of obtaining AAV variants with…

Drug resistance is a leading cause of cancer death in children and adults with leukemia. St. Jude Children's Research Hospital scientists have developed a novel strategy to find the genes responsibleCredit: St. Jude Children's Research…

Data support nuclear hormone receptor gene NR2E3 as genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases and potentially serve as a broad-spectrum therapyCredit: Ocugen, Inc. MALVERN, PA, March 3, 2020…

Credit: Image courtesy of Thomas Gaj, University of Illinois CHAMPAIGN, Ill. -- With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an…

A pioneer in the field, CHOP-based Center brings together researchers, physicians, and genetic counselors to help patients navigate the complexities of genetic testingCredit: Children's Hospital of Philadelphia Philadelphia, February…

Mechanisms of carcinogenesis and tumor progression The RAF protein could be a therapeutical target to treat the tumor growth in regulated pathways by the p38 protein, according to a new study published in the journal Scientific Reports by…

Credit: Eyleen de Poel en Maarten Geurts, copyright UMC Utrecht and Hubrecht Institute Utrecht, The Netherlands, 20th of February 2020. While the genome editing tool CRISPR/Cas9, developed in 2012, cuts a mutation out of a gene and…