Sentences with phrase «gene therapy approach»
But such gene therapy approaches in humans are notoriously difficult.
smafoundation.org
My laboratory utilizes gene therapy approaches to deliver treatments to the eye.
St. Jude is developing innovative gene therapy approaches for patients with blood diseases, immune disorders and other conditions.
«Efficacy of new gene therapy approach for toxin exposures shown in mouse study.»
According to Dunbar and her colleagues, these methods, including the CRISPR / Cas9 approach, would provide ways to correct or alter an individual's genome with precision, which should translate into broader and more effective gene therapy approaches.
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.
The new study ups the ante on a similar gene therapy approach with natural antibodies that 6 years ago showed promise in monkey experiments, says an accompanying Nature editorial by AIDS vaccine researcher Nancy Haigwood of Oregon Health & Science University in Beaverton.
Most current gene therapy approaches rely on cold viruses, crippled so that they can't multiply within the body, to insert copies of potentially therapeutic genes into cells.
The successful results obtained with ADA - SCID provided a rationale for extending the HSC gene therapy approach to other diseases.
Because chronic heart failure in humans is accompanied by a reduction in the number of myocardial beta - adrenergic receptors and in inotropic responsiveness, these results suggest a potential gene therapy approach to this disease state.
For diseases that may be caused by mutations in many different genes, developing individual gene therapy approaches can be prohibitively costly and time - intensive to pursue.
«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.»
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.
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.
In the current review we will present the molecular pathways of CRC and discuss novel gene therapy approaches.
Therapeutic stimulation of cardiac lymphangiogenesis — protein vs. gene therapy approaches post-MI Ebba Brakenhielm, Rouen University
St. Jude researchers are now focused on applying investigational gene therapy approaches to treat X-linked SCID, hemophilia B and a related disorder, hemophilia A.
However, many cancer patients treated with rapamycin analogues do not respond and this makes the use of mTOR siRNA an interesting alternative gene therapy approach, at least from the theoretical point of view.
An AAV gene therapy approach has shown in preclinical in vivo models to produce stable gene expression, enhanced survival, and prevention of hypoglycemia.
Furthermore, Bennett added, the research holds promise for using a similar gene therapy approach for other retinal diseases.
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.
... But Translation from Lab to OR Poses Many ChallengesSeveral research groups are pursuing gene therapy approaches to regenerate skin, such as using genes to control expression of growth factors involved in skin healing.
These studies will contribute to improve current gene therapy approaches and advance patients» care.
This is the first time the agency has greenlighted a gene therapy approach for use in the United States.
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.
Dr. Steven R. Goodman, Editor - in - Chief of Experimental Biology and Medicine said «The study by Qi and colleagues is very exciting as it combines tissue engineering and gene therapy approaches to successfully repair defective articular cartilage.
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.
A patent application has been submitted by Inserm Transfert for this gene therapy approach.
The emergency treatment would be the first test of their gene therapy approach over such a large and severely damaged area.
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.
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.
This gene therapy approach restored normal blood glucose levels in diabetic mice for an extended period of time, typically around four months, and the new insulin - producing cells derived almost exclusively from alpha cells.
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.
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.
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).
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.
Dominic Eberle (Ader, TUD)-- «Cell transplantation and gene therapy approaches for the treatment of retinal degenerative disorders» (2012)
«For them, the gene therapy approach may eventually offer another option.»
Genetic treatment of a molecular disorder: gene therapy approaches to sickle cell disease.
In addition to ADA - SCID and WAS, the research group is currently investigating the molecular and cellular mechanisms linked to mutations in genes encoding the gp91phox NADPH oxidase (Chronic Granulomatous Disease), ARPC1B and ADA2 / CECR1 with the final goal to develop a gene therapy approach for these diseases.
GSD1a is an area of significant unmet medical need, where a gene therapy approach has the potential to replace the defective gene and significantly improve treatment for children.
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.