Rice wins fed grant to advance sickle cell disease therapy
Bioengineer Gang Bao studies genome-editing outcomes to enable a future clinical trialCredit: Jeff Fitlow/Rice University HOUSTON - (April 24, 2020) ...
Tag: Gene Therapy
Sanfilippo C syndrome: New brain cell models to evaluate therapies
Fighting degenerative diseasesCredit: UNIVERSITY OF BARCELONA - CIBERER The Sanfilippo syndrome type C is a severe neurodegenerative disease which appearws ...
University of Guelph develops effective way to replenish threatened plants
University of Guelph researchers used advanced cloning techniques to give the threatened Hill's thistle a fighting chance.Credit: University of Guelph ...
Researchers map mechanism to explain role of gene mutations in kidney disease
Study could pave the way for future targeted treatments for kidney diseaseCredit: University of Maryland School of Medicine Researchers from ...
Inherited mutation can predispose children to a type of brain tumor
Collaboration co-led by researchers at St. Jude Children's Research Hospital discovers a novel predisposition gene in pediatric medulloblastomaCredit: St. Jude ...
New 3D cultured cells mimic the progress of NASH
Credit: T. Usui / TUAT A research team led by scientists from Tokyo University of Agriculture and Technology (TUAT), Japan, ...
Cancer research, the guardian of the genome has a new ally
A team of researchers of the University of Trento focused on a new molecular mechanism that works like a 'switch'Credit: ...
Benefiting from the national gene vector biorepository
Credit: Mary Ann Liebert, Inc., publishers New Rochelle, NY, March 26, 2020--Gene therapy investigators can greatly benefit from the resources ...
Scientists identify gene that first slows, then accelerates, progression of ALS in mice
Columbia study underscores complexities of deadly neurodegenerative disease; offers promising strategies to target disease's underlying genetic mechanismsCredit: Valeria Gerbino/Maniatis lab/Columbia's ...
New therapeutic strategy against diabetes
Maintaining vitamin D receptor levels in insulin-producing cells could protect against diabetesCredit: CBATEG-UAB Maintaining vitamin D receptor (VDR) levels in ...
Edmonton company Entos developing DNA vaccine against COVID-19
University of Alberta cancer researcher John Lewis's health-care biotechnology company is working on a vaccine to be delivered directly into ...
Tracking adeno-associated virus capsid evolution
Credit: Mary Ann Liebert Inc., publishers New Rochelle, NY, March 18, 2020--Researchers have used high-throughput screening of adeno-associated viral (AAV) ...
St. Jude finds cancer drug resistance genes and possibly how to limit their effects
Drug resistance is a leading cause of cancer death in children and adults with leukemia. St. Jude Children's Research Hospital ...
Nature Gene Therapy publishes preclinical data of Ocugen’s OCU400 genetic modifier
Data support nuclear hormone receptor gene NR2E3 as genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases and ...
New CRISPR base-editing technology slows ALS progression in mice
Credit: Image courtesy of Thomas Gaj, University of Illinois CHAMPAIGN, Ill. -- With a new CRISPR gene-editing methodology, scientists from ...
Roberts Individualized Medical Genetics Center outlines centralized genetic testing model
A pioneer in the field, CHOP-based Center brings together researchers, physicians, and genetic counselors to help patients navigate the complexities ...
Curing genetic disease in human cells
Credit: Eyleen de Poel en Maarten Geurts, copyright UMC Utrecht and Hubrecht Institute Utrecht, The Netherlands, 20th of February 2020. ...
Plant-based relatives of cholesterol could give boost to gene therapy
Credit: Image provided by Gaurav Sahay, OSU College of Pharmacy. PORTLAND, Ore. - Gene-infused nanoparticles used for combating disease work ...
Gene therapy prevents disorders with alcohol exposure in ALDH2 deficiency
Credit: Mary Ann Liebert, Inc., publishers New Rochelle, NY, February 12, 2020--A new study has shown that gene therapy to ...
IDIBELL and Dexeus Mujer will study embryonic development by human embryo genome edition
The team of Dra. Anna Veiga, who leads the study, will optimize the CRISPR/Cas9 technique to modify the human embryo ...
