• Gene therapies in mice have been effective to some extent in treating HIV infection, nicotine addiction, blindness and a fatal nervous system disease called spinal muscular atrophy. (dailypress.com)
  • SILVER SPRING, Md. , May 24, 2019 /PRNewswire/ -- The U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. (prnewswire.com)
  • Utilizing gene therapy methods to probe the genetic requirements to prevent spinal muscular atrophy. (umsystem.edu)
  • Clinical trials investigating drugs that target specific genetic alterations in tumors are important for promoting personalized cancer therapy. (nih.gov)
  • Altogether, our approach provides new insights about design principles for combination therapy in the context of intratumoral diversity, data that should inform the development of drug regimens superior for complex tumors. (aacrjournals.org)
  • For years, astrocytomas have been lumped together based on similar appearance to pathologists studying their structure, cell shape, and other factors," said senior author Adam C. Resnick, PhD. "But our current discoveries show that the genetic and molecular structure of tumors provides more specific information in guiding oncologists toward customized treatments. (oncologynurseadvisor.com)
  • Today, clinicians generally rely on tests that generate genetic profiles of biopsied tumors in bulk rather than one cell at a time. (ucsf.edu)
  • Accelerating the discovery of targeted cancer therapies requires defining the targets present in individual tumors, and there are two main ways to do this, David B. Solit, MD , told participants at the inaugural OncoSET Symposium: Emerging Approaches to Precision Medicine in Chicago. (ascopost.com)
  • In this regard, substantial efforts to enhance the efficacy and safety of cell therapy are continuously growing along several fronts, including modifications to improve the reprogramming efficiency of inducible pluripotent stem cells (iPS), genetic engineering of adult stem cells, and administration of growth factors or small molecules to activate regenerative pathways in the injured heart. (nih.gov)
  • Elucidating the genetic pathways and molecular networks that are perturbed in t-MNs, may facilitate the identification of therapeutic targets that can be exploited for the development of urgently-needed targeted therapies. (ebscohost.com)
  • The Cure Sickle Cell Initiative will take advantage of the latest genetic discoveries and technological advances to move the most promising genetic-based curative therapies safely into clinical trials within five to 10 years. (nih.gov)
  • Sickle cell disease results from a single genetic mutation that causes a person's red blood cells to form an abnormal, sickle shape. (nih.gov)
  • Decades of basic research on sickle cell disease have laid the groundwork for novel genetic approaches to cures, such as the genetic editing of bone marrow cells, which have shown great promise in animal models and in some small scale human studies. (nih.gov)
  • The Cure Sickle Cell Initiative seeks to develop cures for a far broader group of individuals with the disease, and it is initially focusing on gene therapies that modify the patient's own hematopoietic stem cells (HSCs), which make red and other blood cells. (nih.gov)
  • Genetic therapies may use gene transfer or genome editing approaches to change the DNA in a patient's cells to treat a condition. (nih.gov)
  • Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice. (eurekalert.org)
  • This is the first time that genome editing, which precisely targets and repairs a genetic defect, has been done in a living animal and achieved clinically meaningful results. (eurekalert.org)
  • Our research raises the possibility that genome editing can correct a genetic defect at a clinically meaningful level after in vivo delivery of the zinc finger nucleases," said the study leader, Katherine A. High, M.D., a hematologist and gene therapy expert at The Children's Hospital of Philadelphia. (eurekalert.org)
  • The vast majority of genetic variants in the human genome remain uncharacterized. (medicalxpress.com)
  • Genetic engineering also makes it possible to customize one's genome at will. (eupedia.com)
  • Results : Ultra-deep sequencing determined a total of more than 10 million nucleotides of the HCV genome, corresponding to a mean of more than 1000 clones in each specimen, and unveiled extremely high genetic heterogeneity in the genotype 1b HCV population. (nii.ac.jp)
  • Unlike retroviruses, adenoviruses are not integrated into the host cell's genome, so within a few weeks the cell will discard the foreign genetic material, and while both these types are capable of targeting specific types of cells, adenoviruses are able to infect both diving and non-dividing cell types effectively. (mesotheliomacenter.org)
  • In this project, technological breakthroughs in genome sequencing, genome engineering, and stem cell technologies are used as appropriate for interrogation of disease pathophysiology, and development of cell and animal models aimed at functional understanding and development of therapies for the blindness. (edu.au)
  • To reveal genetic effects against viral clearance or treatment response, four independent groups applied a genome-wide association study (GWAS) to HCV infection. (go.jp)
  • To address this issue, we designed a high throughput experimental system to characterize the functional consequences of 91% of all possible genetic variants of the NUDT15 gene," said senior author Jun J. Yang, Ph.D., of the St. Jude Departments of Pharmaceutical Sciences and Oncology. (medicalxpress.com)
  • Massive amounts of genetic information due to the abundance of HCV variants could be an obstacle to evaluate the viral genetic heterogeneity in detail. (nii.ac.jp)
  • Dr Luca Lotta, senior clinical investigator at the MRC Epidemiology Unit, explains the premises behind a genetic study: "A recent concept that has been emerging is that some naturally occurring genetic variants that are present in the general population can simulate and can mimic the effect of drugs. (pharmaceutical-technology.com)
  • Basically because genetic variants are randomly assigned at conception this is similar to what happens in a randomised control trial, where you may randomise people to receive nothing…or to receive a drug that enhances LPL. (pharmaceutical-technology.com)
  • In all, seven genetic variants that may influence myeloma risk and response were identified with significant differences seen among ethnic populations. (technologynetworks.com)
  • To help them develop new therapies, Lipshutz and his collaborators first sought to better understand how arginase deficiency affects the brain in a way that causes the disease's developmental and neurological symptoms. (ucla.edu)
  • The team first defined and reported in 2006 on three lung cancer molecular subtypes, named according to their genetic pattern - bronchoid, squamoid and magnoid. (unchealthcare.org)
  • The report, 'Understanding Neuromuscular Disease Care,' highlights current gaps in care and opportunities to optimize care and accelerate the emergence of new therapies. (news-medical.net)
  • The collaboration will leverage Affinia Therapeutics' capsid engineering expertise and Vertex's scientific, clinical and regulatory capabilities to accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF). (businesswire.com)
  • Immediate virologic responders (n = 8) showed a significant reduction in the genetic complexity spanning all the viral genetic regions at the early phase of IFN administration (p = 0.037). (nii.ac.jp)
  • These groups almost simultaneously reported a strong association of interleukin (IL)-28B polymorphisms with viral clearance or final decision of HCV therapy. (go.jp)
  • In a story Oct. 9 about an experimental gene therapy for blindness, The Associated Press erroneously reported the deadline for a decision by the U.S. Food and Drug Administration. (times-standard.com)
  • On Thursday, U.S. Food and Drug Administration advisers will consider whether to recommend approval of a gene therapy that improved vision for these three youths and some others with hereditary blindness. (times-standard.com)
  • Scientists at the University of California, San Diego School of Medicine have elucidated a genetic interaction that may prove key to the development and progression of glaucoma, a blinding neurodegenerative disease that affects tens of millions of people worldwide and is a leading cause of irreversible blindness. (healthcanal.com)
  • Thus, early administration could arrest disease progression and even prevent this form of genetic blindness. (arvojournals.org)
  • According to the UC San Diego team, the next step is to conduct preclinical studies to assess the efficacy and safety of antisense oligonucleotides - strands of synthesized DNA or RNA that can prevent transfer of genetic information - which might inhibit p16INK4a expression and prevent worsening of glaucoma. (healthcanal.com)
  • NHLBI has named hematologist Edward J. Benz Jr., M.D., President and CEO Emeritus of Dana-Farber Cancer Institute, as the Initiative's executive director, and the Emmes Corporation, a contract research organization with specialized expertise in clinical trials, gene and cell therapy development in preclinical studies, as its coordinating center. (nih.gov)
  • Further developments in gene therapy would allow the progression and optimization of future applications regarding vector tools, therapeutic constructs, delivery systems, design of preclinical evaluation and clinical trials. (frontiersin.org)
  • In poor CYP2C19 metabolizers who are undergoing percutaneous coronary intervention for acute coronary syndromes, ticagrelor (Brilinta) or prasugrel (Effient) should be considered as an alternative to clopidogrel (Plavix) for antiplatelet therapy. (aafp.org)
  • The objective of the RAPID GENE study is to evaluate the feasibility, efficacy and safety of a pharmacogenomic approach to anti-platelet therapy following coronary artery stenting using a CYP2C19*2 point-of-care genetic test. (clinicaltrials.gov)
  • Effective medical treatment following acute coronary syndromes and percutaneous coronary intervention (PCI) consists of dual anti-platelet therapy with aspirin and clopidogrel. (clinicaltrials.gov)
  • So far the new LPL enhancer drugs remain in the pre-clinical and early clinical stages of development and no large clinical trials have been carried out to demonstrate LPL enhancers' efficacy in combination with cholesterol lowering agents, but this study is a vital step in exploring the potential of combination therapies for coronary and other conditions. (pharmaceutical-technology.com)
  • Through this initiative, NHLBI seeks to support the development of cell and genetic therapies resources, clinical trials, comparator analyses of different management strategies, data repositories and resources, and patient and advocate engagement activities related to curative therapies for this condition. (nih.gov)
  • Scientists and clinicians at Children's Hospital Los Angeles in California have developed a comprehensive genetic testing panel to detect genetic markers that respond to target therapies, according to new research published in the New England Journal of Medicine . (integrativepractitioner.com)
  • The quantification of genetic markers for oligodendrocyte progenitor cells (OPCs) and mature oligodendrocytes revealed reduced differentiation of OPCs into oligodendrocytes in mct8 −/− larvae and adults. (biologists.org)
  • Cite this: FDA Approves Carglumic Acid Therapy for Rare Genetic Disorder - Medscape - Mar 19, 2010. (medscape.com)
  • CALGARY - A big sushi meal would have once made Josh McQuillin gravely ill, but the British Columbia man can now gorge on one of his favourite foods worry-free thanks to a clinical trial for his rare genetic disorder. (lfpress.com)
  • Dr Nierman provides a quick introduction to the human karyotype (the organized profile of a person's chromosomes) to frame our understanding of genomic therapy. (onlineethics.org)
  • Different subtypes among HIV-1 viruses impact disease progression differently [ 15 ] and may also have differential sensitivity to antiretroviral therapy (ART) drugs [ 16 ]. (hindawi.com)
  • Gene therapy involves the replacement or modification of a genetic variant to restore or enhance cellular function or the improve response to nongenetic therapies. (ama-assn.org)
  • Genetic engineering involves the use of recombinant DNA techniques to introduce new characteristics or traits. (ama-assn.org)
  • The gene therapy involves an immune system cytokine, either Interferon gamma (IFNγ) or MDA-7/IL- 24 - the latter discovered by Dr. Fisher and works by inducing metastatic cells to commit cell suicide (apoptosis). (nfcr.org)
  • Unlike traditionally animal and plant breeding, which involves doing multiple crosses and then selecting for the organism with the desired phenotype, genetic engineering takes the gene directly from one organism and inserts it in the other. (wikipedia.org)
  • In gene therapy, scientists use vector molecules to insert a functional gene into the cells of individuals suffering from a disorder caused by a defective gene. (britannica.com)
  • Khan is leading the Canadian portion of an international clinical trial on gene therapy for urea cycle disorder. (lfpress.com)
  • The WARFARIN Study is a clinical trial designed to determine if the use of genetic information related to warfarin sensitivity can help create a dose of warfarin that will result in less hospitalizations and deaths related to warfarin. (clinicaltrials.gov)
  • CGCs in this field may also implement education tools, review family history, deliver test results, coordinate clinical trial consent/enrollment, and facilitate genetic testing such as HLA typing. (parentsguidecordblood.org)
  • MRC's findings came from a genetic study which aims to simulate a clinical trial that would test the efficacy of the LPL enhancers. (pharmaceutical-technology.com)
  • A normally functioning gene is introduced into a cell where there are genetic anomalies or defects. (supremeessays.com)
  • The vector then unloads its genetic material into the target cell. (scribd.com)
  • Pharmacologic and genetic strategies to enhance cell therapy for cardiac regeneration. (nih.gov)
  • The main aim of this PhD project was to develop a strategy to enhance T cell function for tumour therapy. (bl.uk)
  • Cell therapy using multiple cell types, including cord blood, is currently in various stages of clinical research and development. (parentsguidecordblood.org)
  • CGCs also talk to families that are considering clinics which offer stem cell therapies without regulatory oversight or the ability to collect clinical data regarding treatment outcomes. (parentsguidecordblood.org)
  • Affinia Therapeutics' innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and we're excited to partner with them to bring together their technology platform with our research and development expertise. (businesswire.com)
  • Myoblast fusion in has turned into a powerful genetic system with which to unravel the mechanisms underlying cell fusion. (exposed-skin-care.net)
  • This cellular diversity, however, is what makes cancer so difficult to treat, as each type of cell in a tumor responds differently - and sometimes not at all - to cancer therapies. (ucsf.edu)
  • Genetic engineering does not normally include traditional breeding, in vitro fertilisation, induction of polyploidy, mutagenesis and cell fusion techniques that do not use recombinant nucleic acids or a genetically modified organism in the process. (wikipedia.org)