«Efficacy of new
gene therapy approach for toxin exposures shown in mouse study.»
Dominic Eberle (Ader, TUD)-- «Cell transplantation and
gene therapy approaches for the treatment of retinal degenerative disorders» (2012)
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 att
gene 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 att
Gene 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 dec
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
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 dec
gene 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 g
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 g
gene 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.