Sentences with phrase «gene therapy approaches for»

Dominic Eberle (Ader, TUD)-- «Cell transplantation and gene therapy approaches for the treatment of retinal degenerative disorders» (2012)

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

Not exact matches

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 permanent gene therapy method could be a nonsurgical contraceptive approach for pets or stray animals.

This approach may make gene therapy a viable method for delivery of polypeptides that require rapid and regulated delivery.

«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.»

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.

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

Despite this new direction for identifying targets for pharmaceutical interventions against RP, the researchers underscore that gene therapy still has great potential and possible benefits, and they are actively pursuing efforts with this approach for several forms of RP.

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.

Most approaches for regenerative therapy for the heart use stem cells or gene therapy.

«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.»

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.

The nanotechnology approach that is being tested for inhibitor control could also improve the haemophilia treatment that is now at the cutting edge of clinical research: gene therapy.

Existing approaches to delivering DNA into cells for producing these gene therapies include viruses, external electrical fields or harsh chemical reagents.

As the central nervous system is also a target of AAV vectors, Hélène Puccio and Patrick Aubourg's teams are investigating whether a similar approach using gene therapy could be as effective for the spinal cord and cerebellum as it is for the heart.

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.

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.

«Regarding the first part of the question, gene therapy is approaching clinical realization for the treatment of neoplastic and metabolic diseases.

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.

«We recognize that there are many other approaches [to pay for gene therapy] that thoughtful study might uncover,» the authors conclude, «but we need to begin to ensure that economic challenges are given the attention they deserve.»

The next step will be to optimize the approach and to extend it to protein - protein interactions, protein - gene interactions, disease - protein interactions, disease - drug interactions all with a view to improving diagnostics and tailoring therapy for the individual patient based on the outcomes of their personal biological network analysis.

Down the road, gene therapy and other approaches look likely to bring longer - term treatments for patients with the rare bleeding disorder.

Although the study's approach wasn't a sure bet, the researchers hoped to go further than any other gene therapy trial yet for this relatively common inherited disease that fills people's lungs with sticky mucus that promotes deadly infections.

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.

«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.»

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.

Sangamo Biosciences (Nasdaq: SGMO, US) The company is pioneering a new sophisticated approach for treatment of HIV patients by cell gene therapy.

This is followed by a presentation by Dr Alaina Schlinker, Fresenius Kabi, as she discusses practical approaches to addressing some of the key processing challenges for cell and gene therapy products.

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.

Rob Burgess, JAX Center for Precision Genetics - Developing personalized gene therapy approaches

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.

With the aim of pioneering a safe, effective gene therapy approach, St. Jude scientists have spent years developing an innovative vector for X-linked SCID.

«For them, the gene therapy approach may eventually offer another option.»

«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.

Current precision medicine approaches aim to disable those mutated genes, and many cancer patients today have their tumors analyzed for mutated genes that can be treated with available targeted therapies.

Now that Spark has grown and its therapy for the RPE65 gene is approaching an FDA decision, she says, the company is expanding its portfolio, with programs directed at new targets.

Gene therapy is a promising approach for treating a large spectrum of human diseases and particularly monogenic diseases that can be corrected through gene replacement therapy based on introducing a functional copy of the defective gGene therapy is a promising approach for treating a large spectrum of human diseases and particularly monogenic diseases that can be corrected through gene replacement therapy based on introducing a functional copy of the defective ggene replacement therapy based on introducing a functional copy of the defective genegene.

And he should know - Wilson has been studying CF for over 20 years, as well as many other rare diseases and gene - therapy approaches to treat them.

The successful results obtained with ADA - SCID provided a rationale for extending the HSC gene therapy approach to other diseases.

Activators of histone acetyltransferases (HAT) provide an alternative approach for the selective activation of gene expression, however little is known about the potential of HAT activators as drug therapies for PD.

Clinical Applications and Bioprocessing Strategies Fundamentally, gene therapy should exceed the efficacy of existing therapeutic approaches and provide therapeutic strategies for unmet medical needs.

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.

Scalable Purification of Viral Vectors for Gene Therapy: An Appraisal of Downstream Processing Approaches

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.»

Sophisticated cell targeting systems such as the gene therapy approach developed for senescent cell clearance by Oisin Biotechnologies could also be turned to stem cell or immune cell destruction, given suitable markers of cell chemistry.

This approach may also be applicable to a number of rare genetic diseases caused by splicing defects, including ataxia telangiectasia, congenital disorder of glycosylation, and Niemann - Pick disease type C. Although the three DMD studies referenced here represent a great step forward for CRISPR gene therapy, it's important to realize that DMD is a simpler case than other genetic diseases we'd like to treat with CRISPR.