Sentences with phrase «gene therapy approaches in»

Lysogene collaborates with University of Massachusetts Medical School (UMMS) for pre-clinical efficacy studies of innovative AAV - based gene therapy approaches in the GM1 gangliosidosis mouse model.

But such gene therapy approaches in humans are notoriously difficult.

Researchers at the 12th International Neuroscience Winter Conference will explore, among other hot topics in neuroscience, the neurobiology of courtship, new gene therapy approaches in Parkinson's disease, the role of sleep in neuropsychiatric disorders and breakthroughs in brain repair.

They currently have a trial under way at the U-M Health System which tests a two - part gene therapy approach in patients with brain tumors called gliomas in an effort to get the immune system to attack the tumor.

Not exact matches

«Today's approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide - range of challenging diseases,» FDA Commissioner Scott Gottlieb said in a statement.

Katherine High, Spark's president and chief scientific officer, expressed her enthusiasm for the early clinical data related to SPK - 8011: «The encouraging start of our SPK - 8011 clinical trial reinforces the strength of our gene therapy platform, delivers human proof - of - concept in a second liver - mediated disease — a significant achievement in the gene therapy field — and positions us well to potentially transform the current treatment approach for this life - altering disease with a one - time intervention.»

Gene therapy delivered to a specific part of the brain reverses symptoms of depression in a mouse model of the disease — potentially laying the groundwork for a new approach to treating severe cases of human depression in which drugs are ineffective.

The investigators caution the approach is years away from use in humans, but gene therapy carries the promise of restoring hearing in people with several forms of both genetic and acquired deafness.

However, they determined the blood levels of SMN are not altered when SMN levels are altered in motor neurons using a gene therapy approach delivered directly into the cerebrospinal fluid to target the central nervous system.

«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.»

Now a paper in the November 2009 issue of Science suggests that the long - sought cure may come from gene therapy — a famously hyped approach to treatment that tragically caused the death of a teenage experimental subject in 1999.

Another candidate, AVXS - 101 (AveXis), is a gene therapy approach to increase SMN levels, which is in Phase I clinical trials.

The benefit of this approach over other gene therapy techniques is that the new method can permanently correct the «defect» in a gene rather than just transiently adding a «functional» one, said Dr. Eric Olson, Director of the Hamon Center for Regenerative Science and Medicine at UT Southwestern and Chairman of Molecular Biology.

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.

With this in mind, independent teams led by Scott and Herzog took the conventional viral - vector approach to inducing tolerance through gene therapy.

Indeed, exposure of the protein produced by the nanoparticle - based gene therapy to the gut mucosa prevents inhibitor development and restores clotting - factor activity in mouse models of both haemophilia A and B. «This approach really could hold big benefit for patients,» says Jörg Schüttrumpf, a transfusion - medicine specialist who led one of the studies performed at the German Red Cross Blood Donor Service in Frankfurt.

His team's approach is based on gene therapy, where a «tame» virus is harnessed to transfer a gene into target cells in the recipient.

«Today's approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide - range of challenging diseases,» FDA Commissioner Scott Gottlieb said in a statement.

A study published January 4th in Cell Stem Cell demonstrates that a gene therapy approach can lead to the long - term survival of functional beta cells as well as normal blood glucose levels for an extended period of time in mice with diabetes.

Field reports suggest that not all K13 mutations are capable of causing resistance, and the genetic system developed by Dr. Fidock to study K13, based on DNA repair approaches that are being used in human gene therapy studies, will be critical in identifying real hot spots of resistance.

The team envisions that in the future, deaf people might benefit from gene therapy similar to the approaches currently being tested in clinical trials for other diseases.

«Because this mutation already exists in nature and is benign, this «organic gene therapy» approach should be effective and safe to use to treat, and possibly cure, serious blood disorders.

A bone marrow transplant is the only standard treatment, but the gene therapy approach, developed by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, offers a new line of attgene therapy approach, developed by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, offers a new line of therapy approach, developed by researchers at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy, offers a new line of attGene Therapy in Milan, Italy, offers a new line of Therapy in Milan, Italy, offers a new line of attack.

Researchers found the gene therapy approach had stronger results when used in combination with either depletion of immunosuppressive cells from the tumor mass or with immune checkpoint blockade.

The research team found that when a particular mitochondrial fission protein (GTPase dynamin - related protein - 1 — Drp1) was blocked using either gene - therapy or a chemical approach in experimental models of PD in mice, it reduced both cell death and the deficits in dopamine release — effectively reversing the PD process.

«Engineering novel methods of delivering monoclonal antibodies could be an important approach in the fight against infection and in unique treatment situations,» said senior author David B. Weiner, PhD, a professor of Pathology and Laboratory Medicine and chair of the Gene Therapy and Vaccine Program.

Several research groups are pursuing gene therapy approaches to regenerate skin, such as using genes to control expression of growth factors involved in skin healing.

Fisher and his colleagues are pioneering this approach and have already reported success in preclinical experiments utilizing UTMD technology and mda - 7 / IL - 24 gene therapy in prostate and colorectal cancer models.

This approach for gene therapy to treat fatty liver disease, for example, might prove both safer and more effective than reengineering cells in the liver itself.

The new findings, published online in the journal Molecular Cancer Therapeutics, show that combining rapamycin with a gene therapy approach enhanced the animals» ability to summon immune cells called CD8 + T cells to kill tumor cells directly.

«RNAi therapies are a unique approach to cancer treatment as they have the potential to «turn off» the genes» coding for proteins involved in cancer cell division,» said Ramesh K. Ramanathan, M.D., medical director of the Virginia G. Piper Cancer Center Clinical Trials Program at Scottsdale Healthcare and deputy director of the Clinical Translational Research Division of the Translational Genomics Research Institute (TGen) in Phoenix, Ariz. «Using a lipid nanoparticle, the RNAi drug can be delivered to a cancer cell to block the expression of specific proteins involved in tumor growth.»

«Efficacy of new gene therapy approach for toxin exposures shown in mouse study.»

PNP Therapeutics, Inc. — a company based on a gene - therapy technology developed by scientists at Southern Research Institute and the University of Alabama at Birmingham (UAB)-- has found an approach that has abolished otherwise unmanageable human cancers in preclinical rodent studies.

Gene Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decGene Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a Therapy Approaches Studied for «Almost Every Tissue»... Dr. Giatsidis and coauthors reviewed the state of the art in research on gene therapy techniques for treatment of local disorders and injuries — the first such review in more than a decgene therapy techniques for treatment of local disorders and injuries — the first such review in more than a therapy techniques for treatment of local disorders and injuries — the first such review in more than a decade.

The collaboration will combine Lysogene's outstanding translational and clinical expertise in gene therapy for CNS disorders with the unique preclinical expertise and infrastructure of UMMS and AU to design and test innovative AAV - based gene therapy approaches to treat GM1 - gangliosidosis.

While the vast majority of research in this field is focused on developing stem cell, gene and tissue engineering therapies, these approaches have not yet delivered on their promise despite nearly two decades of research.

This specificity in treatment fits with an emerging approach in cancer treatment nationwide, known as personalized medicine, in which the therapies for each patient are selected based on the genes altered in their tumors.

The latest developments in CAR - T translation and manufacturing including alternate gene transfer methods to deliver CARs; novel gene editing approaches, advances in CAR design to improve safety and efficacy, and progress in creating an «off - the - shelf» allogeneic CAR - T therapy.

The finding, published in the current issue of Genes and Development1 represents a different approach to cultivating specialized cells for transplant therapy than that being pursued in other labs.

«In our study, we used a gene therapeutic approach, but you could purify the modified protein and inject it intravenously just like it is already done for enzyme replacement therapy,» explains Spencer.

Alta Charo, a professor of law and bioethics at the University of Wisconsin at Madison, reviewed the different approaches that countries have taken in trying to regulate gene therapy.

Oisín Biotechnology — a startup with initial funding from SENS Research Foundation, the Methuselah Foundation, and others — is close behind them, having developed a form of gene therapy that — in animals at least — also destroys these cells, using an approach that is less inherently likely to destroy healthy cells along with senescent ones.

«Our breakthrough findings in psoriasis underscore the unique power of our population approach for tackling the genetics of just about any common condition, and we hope to push this program forward to identify the gene involved as well as targets for the development of new therapies,» said Dr. Kari Stefansson, CEO of deCODE genetics.

An AAV gene therapy approach has shown in preclinical in vivo models to produce stable gene expression, enhanced survival, and prevention of hypoglycemia.

Many of the most promising approaches fall into four categories: the retinal prosthetic, gene therapy, stem cell treatments, and a technique that uses optogenetics, a way to engineer nerves to fire in response to bursts of light.

Conversely, by using a gene therapy approach to elevate progranulin levels, scientists were able to prevent these abnormalities and block cell death in this model.

Despite the marginal improvements in survival of patients suffering from malignant glioma treated with gene therapy vectors, the clinical trials conducted so far using viral vectors, in particular adenoviral vectors, have proven that the use of adenoviral vectors is a safe therapeutic approach, even in large, multicenter, phase 3 clinical trials.

Like many scientists, we think that gene silencing is the most promising approach to developing meaningful therapies for HD families, and it's gratifying that large companies are willing to make a large financial investment in the therapy.

Van Meir cautions: «We need to have a better understanding of all the genes that are turned on or off by silencing MBD2 in a given cancer before we can envision to use this approach for therapy.»

Gene therapy a therapeutic approach in which engineered genes are introduced into a patient's cells to treat a disease.