Sentences with phrase «developing gene therapies»

The results suggest caution when interpreting data from molecular biological studies or developing gene therapies to treat various diseases.

U.S. drug giant Pfizer and California - based biotech Sangamo Therapeutics are partnering to develop a gene therapy to treat ALS, or Lou Gehrig's disease.

Michael Kaplitt, a neurosurgeon at Weill Cornell Medical College in New York, whose lab develops gene therapies for brain disorders, teamed up with Greengard and other colleagues in the new study.

The finding offers potential for developing both gene therapy and more effective protein replacement treatments for hemophilia A, the most common form of hemophilia.

A team of researchers at the Stanford University School of Medicine has used a gene - editing tool known as CRISPR to repair the gene that causes sickle cell disease in human stem cells, which they say is a key step toward developing a gene therapy for the disorder.

More and more, Sweeney says, the immune system is proving to be the most difficult hurdle in developing gene therapy for humans.

Noting the potential of gene therapy to be a one - time treatment for rare and serious diseases that otherwise cost hundreds of thousands, if not millions, of dollars in chronic care over a lifetime, Stuart Orkin, MD, and co-author Philip Reilly, MD, JD, of Third Rock Ventures, seek to «catalyze the discussion» by suggesting several new models for valuing, pricing and developing gene therapy.

One of those could be a widely applicable drug or nutritional supplement, says Tsang, who has been involved in developing gene therapy for treating eye diseases.

Researchers from the Institute of Neuroscience at the Universitat Autònoma de Barcelona have discovered the cellular mechanism involved in memory consolidation and were able to develop a gene therapy which reverses the loss of memory in mice models with initial stages of Alzheimer's disease.

The research suggests the possibility, Kahn explains, of developing gene therapy treatments using fat cells that aid in treating metabolic diseases, cancer or other conditions in the liver or other organs.

Use of this model to better understand the roles of the anaphylatoxins and the MAC, and to develop a gene therapy for AMD, is truly groundbreaking.

We recently developed such an animal model and propose to use it to study the role of complement in AMD as well as develop a gene therapy.

Our lab is developing gene therapy strategies to treat optic neuropathies, potentially blinding diseases of the optic nerve.

He is one of the world's leading authorities in gene therapy, having developed a gene therapy vector, based on a stripped - down version of HIV, that successfully delivers genes for therapeutic purposes.

Developing a Gene Therapy Against Complement Factors in AMD Rajendra Kumar - Singh, PhD Tufts University (Boston, MA)

Researchers at the Biophysics Department of the Federal University of São Paulo (UNIFESP) in Brazil are developing a gene therapy for mucopolysaccharidosis type I...

Dr. Komáromy studies the molecular causes of inherited eye diseases in dogs and is working to develop gene therapies to stop vision loss.

Not exact matches

A Glaxo vet heads over to Genenta to help develop new gene therapy.

• Myonexus Therapeutics, a New Albany, Oh. - based clinical - stage biotechnology company developing transformative gene therapies for limb - girdle muscular dystrophies, raised $ 2.5 million in seed funding.

«We can look at the genes that are abnormal in brain tumors [and] develop therapies to attack those genes.»

US - based pharmaceutical company Gilead Sciences entered the chimeric antigen receptor (CAR) T - cell therapy business through its acquisition of Kite Pharma, and Australian biopharma company CSL Behring acquired US - based Calimmune, a company that develops clinical - stage gene therapy solutions.

«My team at Nationwide Children's has worked with commitment and dedication to develop a therapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Chiltherapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide ChilTherapy at Nationwide Children's.

«While much work remains to be done to develop this research into clinical applications, our findings could have a promising translational impact, both for protein replacement and gene therapy,» said Arruda.

AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXStherapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXSTherapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101.

Gene therapy offers a shortcut to bulking up: At the University of Pennsylvania, H. Lee Sweeney is developing a way to turn the liver into a factory that churns out a muscle growth promoter called myostatin propeptide.

«Fibroblast growth factor receptor inhibitors are new therapies being developed in clinical trials for patients whose cancer cells have genetic alterations in this family of genes,» says Roychowdhury, a member of the OSUCCC — James Translational Therapeutics Program.

The researchers are working with the company Oxford BioMedica to develop this idea into a gene therapy for cancer.

In science news around the world, NASA's Cassini mission is about to take its final plunge into the atmosphere of Saturn after 13 years providing an unprecedented view of the planet and its moons, a fight over whether to preserve or develop of one Europe's oldest gold mining sites heats up again, the U.S. Food and Drug Administration approves the first cancer gene therapy for people, a U.S. court gives a green light to a $ 1 billion lawsuit brought by the Guatemalan victims and survivors of mid — 20th century syphilis experiments by research institutions including Johns Hopkins University, and more.

«The challenge has been how to direct certain therapies designed to manipulate genes of interest in specific cells without developing a specific drug carrier for each specific cell type.

Researchers are developing molecular switches that can inactivate transplanted genes, paving the way for safer gene therapies.

Moreover, researchers have already developed a potential therapy that targets the gene's protein.

«If this approach works in humans, it will really change the conversation that providers have with patients,» Scadden said, especially for those «who have these underlying genetic disorders and for who the new gene - editing and gene therapy techniques are being developed.»

«Stem cell gene therapy could be key to treating Duchenne muscular dystrophy: Approach developed at UCLA holds promise for 60 percent of patients with the deadly disease.»

Two of 10 children treated with gene therapy for SCID in a French trial develop leukemia, researchers announced in 2002, and it is discovered that the virus had inserted genes in several unexpected places around the genome, leading the cells to become cancerous.

For his part, Collins, who has led NIH since 2009 and been kept on by the Trump administration, pointed to an array of promising NIH activities, including the development of new technologies to provide insights into human brain circuitry and function through the Brain Research through Advancing Innovative Neuroethologies (BRAIN initiative) and the use of the gene - editing tool CRISPR - Cas9 to correct mutations and clear the way to develop and test a «curative therapy» for the first molecular disease: sickle cell disease.

When conventional wisdom said only gene therapy could correct the root cause of cystic fibrosis, Vertex developed pill - based treatments that address the underlying disease mechanism, says Altshuler.

«Switching off a gene is of course feasible, the issue is whether it will have the desired effect and whether it's the right thing to do... But in terms of trying to develop a therapy to delay or prevent hair graying, that is something that is potentially feasible; yes.»

«This is a seminal step in identifying key pathways and molecules involved in kidney cancer so that specific therapies that target these new genes can be developed to treat this cancer.»

Washington University chemist Karen L. Wooley created Knedels, synthetic nanometric cagelike polymer structures that mimic viruses and could be developed into a safer delivery system for gene therapy.

Despite much effort, no one has managed to develop an effective form of gene therapy to treat the lung problems of people with cystic fibrosis.

Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and Houston Methodist have developed an alternative treatment in which virus - specific cells protect patients against severe, drug - resistant viral infections.

«Our gene therapy protocol is not yet ready for clinical trials — we need to tweak it a bit more — but in the not - too - distant future we think it could be developed for therapeutic use in humans,» says Jeffrey Holt, PhD, a scientist in the Department of Otolaryngology and F.M. Kirby Neurobiology Center at Boston Children's and an associate professor of Otolaryngology at Harvard Medical School.

«These approaches,» said Sudharsan, «may help sustain photoreceptor cells for the time period needed to develop a specific gene therapy.»

This type of treatment is what we call gene therapy and researchers have been working on developing techniques to accomplish this for many years.

«One of the major limitations in developing a corrective gene therapy is that it will treat only a single disease,» said Sudharsan.

But for a gene therapy to be effective, one must know the precise gene responsible for a given individual's disorder and develop a tailored treatment.

With the data provided by the FLI researchers, scientists around the world may now set targeted mutations to investigate the relevance of single genes for aging, in order to better understand regulating mechanisms and, eventually, develop new approaches for therapy or prevention of aging - related diseases.

But it's much harder to develop therapies that restore malfunctioning genes that should be triggering cell death in abnormal cells, known as tumor - suppressor genes.

«Researchers ID cancer gene - drug combinations ripe for precision medicine: Yeast, human cells and bioinformatics help develop one - two punch approach to personalized cancer therapy.»

But it's still unknown whether the DNA will integrate into chromosomes, says John Polo, a molecular biologist at Chiron Technologies Center for Gene Therapy in San Diego, California, which is developing similar vaccines for HIV and the herpes virus.