English
Arabic
Chinese (Simplified)
Dutch
Esperanto
French
German
Greek
Italian
Japanese
Korean
Portuguese
Russian
Spanish
UrduDisney lab collaboration reawakens heart cells’ silenced VEGF-A healing system by targeting non-coding RNA
Tag: Gene Therapy
Tiny engineered therapeutic delivery system safely solves genetic problems in mice
Nanomaterials show promise for treating hemophilia and cardiovascular disorder
New gene therapy approach eliminates at least 90% latent herpes simplex virus 1
In Nature Communications, Fred Hutch scientists demonstrate in a mouse model how gene therapy could cure an infection that afflicts billions of people
568 genes identified with the potential to trigger cancer
Cancer is a group of diseases characterised by uncontrolled cell growth caused by mutations, and other alterations in the genome of cells. A tumour can present from hundreds to thousands of mutations, but only a few are vital for its…
Nanoparticles enhance gene therapies for eye disease
NIBIB-funded researchers have created nanoparticles for successful gene therapy of a mouse model of macular degeneration. The nanoparticle carriers have the potential to significantly expand the effectiveness of gene therapies for human eye diseases, including blindness.
Engineered capsids for efficient gene delivery to the eye
New Rochelle, NY, August 13, 2020–A rational design approach created novel variants of adeno-associated viral (AAV) capsids. These have improved transduction properties in the mouse retina and cornea. as reported in the peer-reviewed journal Human Gene Therapy . Click here…
First in Human Study with Novel Antisense Oligonucleotide
New Rochelle, NY, August 11, 2020–A single intravenous dose of MRG-110, an anti-microRNA drug, significantly reduced miR-92a levels in the blood of healthy humans. Inhibition of miR-92a has shown beneficial effects in animal models, including improved vascularization after myocardial infarction,…
Scientists replace malfunctioning ‘vacuum cleaner’ cells linked to neurological disorders
Researchers at Fudan University in Shanghai, China have developed three different techniques that successfully replace almost all malfunctioning microglia – each technique with its own advantage in application. The researchers published their findings on Aug 11, 2020 in Cell Reports…
The CNIO pave the way for a future gene therapy to reverse pulmonary fibrosis associated with ageing
Idiopathic pulmonary fibrosis is a potentially lethal disease for which there is currently no cure and that is associated with certain mutations or advanced age. The Telomeres and Telomerase Group at the Spanish National Cancer Research Centre (CNIO) had previously…
Gene therapy targets inner retina to combat blindness
New Rochelle, NY, August 10, 2020–Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease. Adeno-associated viral (AAV)-mediated expression of the human CLN3 gene led to significant survival of bipolar cells…
Engineers enlist fungi to advance against disease
Federal grant helps Rice’s Gao lab find gene-editing strategies to counter drug resistance
Novel approach reduces SCA1 symptoms in animal model
Research has shown that a mutation in the ATAXIN-1 gene leads to accumulation of Ataxin-1 (ATXN1) protein in brain cells and is the root cause of a rare genetic neurodegenerative disease known as spinocerebellar ataxia type 1 (SCA1). How healthy…
Dr. Neville Sanjana receives 2020 Cancer Research Institute Technology Impact Award
Grant supports genome engineering study to discover modifier genes that can boost the effectiveness of CAR T-cell therapy for pancreatic cancer patients
A research team of the CNIO and the HKUST identifies how some gliomas develop chemoresistance
A team led by Massimo Squatrito, Head of the Seve Ballesteros Foundation Brain Tumour Group at the Spanish National Cancer Research Centre (CNIO), have made important findings of how some gliomas can acquire chemoresistance. Carried out together with the laboratory…
Identified gene mutations impact on the severity of a type of hematologic cancer
The Myelodysplastic Syndromes Group of Josep Carreras Leukaemia Research Institute, led by Dr. Francesc Solé, has participated in the first international study that confirms that having two mutated copies of the TP53 gene, as opposed to a single mutated copy,…
Research News Tip Sheet: Story Ideas from Johns Hopkins Medicine
During the COVID-19 pandemic, Johns Hopkins Medicine Media Relations is focused on disseminating current, accurate and useful information to the public via the media. As part of that effort, we are distributing our “COVID-19 Tip Sheet: Story Ideas from Johns Hopkins” every Tuesday throughout the duration of the outbreak.
A versatile vessel for next-gen therapeutics
Harvard’s ARMMs technology to be commercialized by Vesigen Therapeutics for targeted intracellular delivery of large-molecule drugs
Genetic testing could improve screening for osteoporosis
An international team of scientists has developed a novel genetic measure that could dramatically improve how doctors assess the risk of sustaining a fracture due to osteoporosis or fragility A full genome profile can be generated for approximately £35-40 per…
Scientists discover way to stop spread of devastating childhood cancer
Researchers at the University of East Anglia and University of Manchester have made an important breakthrough that could lead to ‘kinder’ treatments for children with bone cancer, and save lives.
Research News Tip Sheet: Story Ideas from Johns Hopkins Medicine
During the COVID-19 pandemic, Johns Hopkins Medicine Media Relations is focused on disseminating current, accurate and useful information to the public via the media. As part of that effort, we are distributing our “COVID-19 Tip Sheet: Story Ideas from Johns Hopkins” every Tuesday throughout the duration of the outbreak.
Scientists Use Nanoparticle-Delivered Gene Therapy to Inhibit Blinding Eye Disease in Rodents
Scientists use nanoparticle-delivered gene therapy to limit blinding retinal disease in rodents.
Massachusetts Eye and Ear Enters Licensing Agreement with Biogen to Develop Treatment for Inherited Retinal Disorder
Massachusetts Eye and Ear, a member hospital of Mass General Brigham, is entering into an exclusive licensing agreement with Biogen to develop a potential treatment for inherited retinal degeneration due to mutations in the PRPF31 gene, which are among the most common causes for autosomal dominant retinitis pigmentosa.
Development of safe liver sinusoid coating agents to increase the efficacy of gene therapy
Controlling the clearance of gene therapy drugs at the liver sinusoidal wall
ISSCR’s first virtual annual meeting opens
More than 1,100 stem cell scientific and industry leaders to share their latest research advances
ISSCR’s first virtual annual meeting opens
More than 1,100 stem cell scientific and industry leaders to share their latest research advances
Discover the latest research in stem cell science during ISSCR 2020 Virtual
The global stem cell event kicks off Tuesday, June 23
All of Us Research Program launches COVID-19 research initiatives
NIH effort expands data collection to shed light on pandemic’s spread and impact
All of Us Research Program launches COVID-19 research initiatives
NIH effort expands data collection to shed light on pandemic’s spread and impact
UCLA receives nearly $14 million from NIH to investigate gene therapy to combat HIV
UCLA researchers and colleagues have received a $13.65 million grant from the National Institutes of Health to investigate and further develop an immunotherapy known as CAR T, which uses genetically modified stem cells to target and destroy HIV.
CICERO software enables detection of cancer-causing gene fusions
St. Jude Children’s Research Hospital software enables detection of previously unknown cancer-causing gene fusions, pointing the way to new treatments.
CICERO software enables detection of cancer-causing gene fusions
St. Jude Children’s Research Hospital software enables detection of previously unknown cancer-causing gene fusions, pointing the way to new treatments.
Researchers model human stem cells to identify degeneration in glaucoma
More than 3 million Americans have glaucoma, a serious eye condition causing vision loss. Using human stem cell models, researchers at Indiana University School of Medicine found they could analyze deficits within cells damaged by glaucoma, with the potential to…
Scientists rescue mini retinas from eye disease via new gene therapy approach
Scientists have developed a new gene therapy approach that offers tremendous promise for one day treating an eye disease that leads to blindness and affects thousands of people across the globe. Researchers from Trinity College Dublin and University College London…
Researchers model human stem cells to identify degeneration in glaucoma
More than 3 million Americans have glaucoma, a serious eye condition causing vision loss. Using human stem cell models, researchers at Indiana University School of Medicine found they could analyze deficits within cells damaged by glaucoma, with the potential to…
Scientists rescue mini retinas from eye disease via new gene therapy approach
Scientists have developed a new gene therapy approach that offers tremendous promise for one day treating an eye disease that leads to blindness and affects thousands of people across the globe. Researchers from Trinity College Dublin and University College London…
City of Hope scientists showcase new findings at the 2020 AACR Virtual Annual Meeting II
These important proof-of-concept studies could one day lead to new real-time diagnostic imaging techniques and the identification of genomic biomarkers that predict treatment results — innovations that will improve health outcomes for cancer patients
City of Hope scientists showcase new findings at the 2020 AACR Virtual Annual Meeting II
These important proof-of-concept studies could one day lead to new real-time diagnostic imaging techniques and the identification of genomic biomarkers that predict treatment results — innovations that will improve health outcomes for cancer patients
Better detection of a type of ovarian cancer could lead to better treatments
Scientists have found that a specific type of ovarian cancer could possibly benefit from existing platinum-based chemotherapy and new DNA repairing treatments, following better testing. The study, led by researchers from RCSI University of Medicine and Health Sciences, is published…
Better detection of a type of ovarian cancer could lead to better treatments
Scientists have found that a specific type of ovarian cancer could possibly benefit from existing platinum-based chemotherapy and new DNA repairing treatments, following better testing. The study, led by researchers from RCSI University of Medicine and Health Sciences, is published…
CSIC researchers will use CRISPR tools to destroy the COVID-19 coronavirus genome
A project aims to use the novel programmable gene editing ‘scissors’ to cut the SARS-CoV-2 RNA genome without altering the rest of the infected cell
A Genethon team has succeeded in inhibiting the immune response linked to AAV
This result opens up new therapeutic prospects and the possibility of treating more patients.
Scientists Aim Gene-Targeting Breakthrough Against COVID-19
Scientists at Berkeley Lab and Stanford have joined forces to aim a gene-targeting, antiviral agent called PAC-MAN against COVID-19.
Gene therapy with a new base editing technique restores hearing in mice
Repairing a single mutation in the Tmc1 gene restored partial hearing in mice
A promise to restore hearing
In a first, researchers use base editing to correct recessive genetic deafness and restore partial hearing
Gene therapy with a new base editing technique restores hearing in mice
Repairing a single mutation in the Tmc1 gene restored partial hearing in mice
A promise to restore hearing
In a first, researchers use base editing to correct recessive genetic deafness and restore partial hearing
Seven Bridges announces international collaboration
Seven bridges announces international collaboration focused on personalized treatment for kids with cancer
CSL Behring and Seattle Children’s Research Institute to advance gene therapy treatments
Initially, the alliance will develop treatment options for patients with two rare, life-threatening primary immunodeficiency diseases — Wiskott-Aldrich Syndrome (WAS) and X-linked Agammaglobulinemia (XLA)
Exceptional stem cell science on tap for ISSCR 2020 Virtual June 23-27, 2020
2020 media registration is open
Exceptional stem cell science on tap for ISSCR 2020 Virtual June 23-27, 2020
2020 media registration is open