Sentences with phrase «for gene therapy studies»
Such long - term expression is important for gene therapy studies in humans.
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The Milto family raised money for a gene therapy study for Batten disease that included their sons Nathan (right) and PJ.
Not exact matches
Abeona Therapeutics (ABEO)- Data for ABO - 102 in MPS IIIA appears encouraging to me (decreases in heparan sulfate, neurocognitive benefits), initial data for ABO - 101 in MPS IIIB showed early promise, EB - 101 in RDEB could see an expedited path to market if the pivotal study yields fruit, and other gene therapy candidates are soon to enter the clinic.
Michael Kaplitt, a neurosurgeon at Weill Cornell Medical College in New York, whose lab develops gene therapies for brain disorders, teamed up with Greengard and other colleagues in the new study.
The study, published online today in Science Translational Medicine, further points to a master switch for these gene sets as a potential target of future therapies.
«I think it awakens the possibility of gene therapy for neuropsychiatric diseases,» says Husseini Manji, a senior investigator at Johnson & Johnson Pharmaceutical Research & Development in Titusville, N.J., who was not involved in the study.
«Combination therapy strengthens T cells in melanoma pre-clinical study: Findings have implications for treating tumors lacking tumor suppressor gene PTEN.»
«One gene closer to regenerative therapy for muscular disorders: Study identifies gene that gets muscle cells to fuse together.»
«Encouraging data for gene replacement therapy for SMA type I, phase 1 study shows.»
This study represents a significant step towards the development of clinical trials in gene therapy for the curative treatment of hereditary deafness and balance loss in humans.
New treatments for spinal cord injury, including stem cells, gene therapy and electrical stimulation, are being studied.
As Saaïd Safieddine, CNRS Director of Research at the Institut Pasteur and co-senior author of the study with Prof. Christine Petit (head of the Genetics & physiology hearing unit at the Institut Pasteur), explains, «we have just shown that it is possible to partially correct a specific form of hereditary hearing loss accompanied by balance problems using local gene therapy performed after the embryogenesis of the ear, which is primarily affected by the mutation responsible for the disorder.
No cases of severe pancreatitis and only one admission to the intensive care unit for an LPLD - related abdominal event were reported in the study published in Human Gene Therapy.
Although these studies did not test for vision restoration, gene therapy in the eye is already starting to be done for other disorders.
Nevertheless,» [the] study is very important because it demonstrates for the first time that we can use gene therapy to transform cells in the brain into ones that will secrete GDNF,» says Jeffrey Kordower, a professor of neurological sciences at Rush Presbyterian Medical Center in Chicago.
Study shows memories formed by the same gene - silencing tool used in embryonic development; a finding could set the stage for new therapies for schizophrenia
These eight strains are all resistant to multi-drug therapy, and were the only ones in the study in which a gene that is responsible for DNA repair is disrupted.
«Gene therapy restores hearing in deaf mice: Proof - of - principle study takes a step toward precision medicine for genetic hearing loss.»
Inhibiting NF - ƘB in microglia in mice slowed disease progression by 47 percent, says Brian Kaspar, MD, a principal investigator in the Center for Gene Therapy at Nationwide Children's and senior author of the new study.
However, Holt's study also showed that gene therapy with TMC2 could compensate for loss of a functional TMC1 gene, restoring hearing in the recessive deafness model and partial hearing in the dominant deafness model.
«What strikes me as innovative here is that you can make the foam almost instantaneously,» says Edith Mathiowitz, a chemist at Brown University in Providence, Rhode Island, who studies such foams for delivering drug or gene therapy.
The study paves the way for CRISPR - Cas9 as a powerful gene editing tool with potential therapeutic applications for inherited diseases — leading to more widely available gene therapy techniques.
The same process has been studied as a potential genetic therapy for more than a decade, because you can target any disease gene with matching dsRNA.
Dorothy Romanus, lead author of the study, states «this analysis supports the value of multiplexed testing for EGFR and ALK gene rearrangements followed by molecularly - guided therapy in decisions surrounding coverage of related testing and targeted therapy.
Wilson famously led a gene therapy study with an adenovirus vector that killed a patient, Jesse Gelsinger, in 1999 — a major setback for the entire field.
«The vectors developed and characterized in this study demonstrate unique and potent biology that justify their consideration for gene therapy applications,» Vandenberghe says.
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.
Dr. Jones obtained his Ph.D. in 2003 from the University of Birmingham Institute of Cancer and Genomic Sciences (United Kingdom) under Professor Lawrence Young, studying the use of gene therapy for targeting Epstein - Barr Virus (EBV) proteins with replication - competent adenoviruses to treat EBV - driven malignancies.
«Even when the genes driving cancer are known, clinicians don't have an efficient way to choose among the hundreds of possible drug therapies,» said study leader Kai Wang, PhD, associate professor of biomedical informatics and director of clinical informatics at the Institute for Genomic Medicine at CUMC.
«Information from this study could help lay the groundwork for therapies that address developmental missteps tied to Hox genes and their regulators.»
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.
«DNA barcoding has the potential to advance the science of selecting nanoparticles for delivering gene therapies,» said James Dahlman, an assistant professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University and the study's principal investigator.
If future studies confirm that role, the genes may become targets for therapies that block myeloma metastasis, she added.
«The study showed for the first time that a modified nNOS gene could be delivered through gene therapy to protect the hearts of mice from Duchenne muscular dystrophy,» said Dongsheng Duan, PhD, co-author of the study and Margaret Proctor Mulligan Professor in Medical Research at the MU School of Medicine.
The team at UF's Powell Center for Rare Disease Research and Therapy conducted the first in - human study of gene therapy to treat respiratory dysfunction in patients with infantile onsetTherapy conducted the first in - human study of gene therapy to treat respiratory dysfunction in patients with infantile onsettherapy to treat respiratory dysfunction in patients with infantile onset Pompe.
«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.»
ASCB President Don Cleveland of the University of California, San Diego, said his team just published an animal study on a gene silencing therapy for treating a form of Lou Gehrig's disease that they now hope to move to clinical studies.
A study investigating the function of the recently discovered enhancer RNA molecules may open new avenues for gene therapy.
«This study highlights genes and molecular processes that could be targets for new therapies to treat peanut - allergy reactions and could be important to understanding how peanut allergy works overall,» said the study's senior author, Supinda Bunyavanich, MD, MPH, Associate Professor, Pediatrics and Genetics and Genomic Sciences at the Icahn School of Medicine at Mount Sinai.
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.
They supported a range of studies, from work on gene therapy tools (including adenovirus and rous sarcoma virus vectors) to development of a new vaccine for the deadly Marburg virus.
«It is a tough calculus, but gene therapy for CF lung disease is still a realistic possibility and studies should be encouraged — especially well - done ones like the U.K. trial.
They found studies using gene therapy to promote the growth of «almost every different tissue» for use in regenerative surgery.
«I've been doing this for more than 40 years and this is one of the most exciting developments we've seen,» says Jerry Mendell, MD, lead author of the study and director of the Center for Gene Therapy at Nationwide Children's.
UPenn gene therapy researcher Jean Bennett, who heads that study (which is separate from Jacobson's) and reported the most dramatic improvements in patients» vision, says those gains seem stable for as long as 7.5 years.
The FDA has also issued warning letters — a less severe sanction — to two of Wilson's collaborators in the study — Steven Raper of the Institute for Human Gene Therapy and Mark Batshaw of Children's National Medical Center in Washington, D.C.
After preclinical studies, a gene therapy trial for SCID - X1 was initiated, based on the use of complementary DNA containing a defective γc Moloney retrovirus — derived vector and ex vivo infection of CD34 + cells.
These are skills that we implement for other international trials of gene therapy for rare genetic diseases of the immune system, blood, muscle, vision or liver... We will continue the current study with the objective of providing treatment for patients.»
Many researchers blamed the therapy for disrupting the patients» DNA, but a new study suggests that the transplanted gene itself may have triggered the cancer.
The results, published in the current issue of Human Molecular Genetics, open the door for pursuing gene editing in nonhuman primates as models for new therapies, including pharmacological, gene - and stem cell - based therapies, said Keith Latham, MSU animal science professor and lead author of the study.