Sentences with phrase «gene therapy approaches»

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.

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

My laboratory utilizes gene therapy approaches to deliver treatments to the eye.

Therapeutic stimulation of cardiac lymphangiogenesis — protein vs. gene therapy approaches post-MI Ebba Brakenhielm, Rouen University

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

Using agent - based modelling and simulation approaches, we contribute to a better understanding of disease development and / or treatment effects (e.g. the context of leukaemia treatment or gene therapy approaches).

But such gene therapy approaches in humans are notoriously difficult.

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.

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 decade.

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.

«Gene therapy approaches fall into two main categories: (1) the transfer of a gene that stimulates the immune system and (2) the insertion of genes within the tumor that sensitizes the tumor to a relatively nontoxic «prodrug.»

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.

The SMA model pig was created using a gene therapy approach by knocking down the levels of pig SMN, followed by treatment with human SMN at early and late time points.

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.

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

So the researchers decided to bypass the gene therapy approach and engineer two stable, longer - lasting forms of GDF15 that they injected directly into the animals.

Such a treatment might be combined with a gene therapy approach, also being pursued by her team and other groups, delivered directly to the brain to curtail mHTT expression.

The emergency treatment would be the first test of their gene therapy approach over such a large and severely damaged area.

Kohn's gene therapy approach using HSC from patient's own blood is a revolutionary alternative to current SCD treatments as it creates a self - renewing normal blood cell by inserting a gene that has anti-sickling properties into HSC.

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.

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 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.

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.

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.

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

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.»

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

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.

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

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.

A gene therapy approach preserves photoreceptor function in a Leber Congenital Amaurosis (LCA) mouse model, even at...

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.

So far, its trials have shown it can improve outcomes when used alongside other multiple myeloma drugs and that could offer it some insulation if the market gets disrupted by new treatment approaches, such as gene therapy.

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.

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.

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.»

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.

Only recently have cell and gene therapy begun to triumph, by borrowing from and blending into each other's approaches.

The proposed strategy reflects a continuing move toward combination therapies, particularly when it comes to epigenetic approaches, which target the mechanisms that control whether genes are switched «on» or «off.»

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.»

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