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Gene therapy in mouse fetuses treats deadly disease

Gene therapy administered in the womb could be used to treat a deadly genetic disease, a study in fetal mice suggests.The results could add to the increasingly popular notion of prenatal gene therapy ... as a way to minimize the damage wrought by some genetic diseases.The US Food and Drug Administration approved the first gene therapy for adults and children last year, and more treatments are crowding

Analysis of Fractional Order Mathematical Model of Hematopoietic Stem Cell Gene-Based Therapy

Hematopoietic stem cell (HSC) has been discussed as a basis for gene-based therapy aiming to cure immune system infections, such as HIV. This therapy protects target cells from infections or ... used to predict the dynamics of HSC gene-based therapy of viral infections. In this paper, we present a fractional mathematical model of HSC gene-based therapy with the fractional order derivative . We

Analysis of Fractional Order Mathematical Model of Hematopoietic Stem Cell Gene-Based Therapy

Hematopoietic stem cell (HSC) has been discussed as a basis for gene-based therapy aiming to cure immune system infections, such as HIV. This therapy protects target cells from infections or ... used to predict the dynamics of HSC gene-based therapy of viral infections. In this paper, we present a fractional mathematical model of HSC gene-based therapy with the fractional order derivative . We

The biological basis and clinical symptoms of CAR-T therapy-associated toxicites

clinical management. Here we review the wide spectrum of effects associated with CAR T cell therapy with a major focus on the pathogenesis of cytokine release syndrome and neurotoxicity as the most common ... ADA-SCID retroviral gene therapy patients. Blood 129, 2624–2635 (2017). ArticlePubMedPubMed Central Google Scholar102. Braun, C. J. et al. Gene therapy for Wiskott-Aldrich syndrome—long-term efficacy

Editorial: Therapy-induced metastasis

The idea for this Special Issue originated from our recent review in Nature Reviews Clinical Oncology entitled “Does the mobilization of circulating tumour cells during cancer therapy cause ... numbers of tumour cells in circulation (CTCs), and potentially increased risk of distant metastasis. This led to our decision to expand the topic by addressing some of the issues associated with therapy

Gene Therapy in the Management of Erectile Dysfunction (ED): Past, Present, and Future

, no viral vectors have progressed to human trials. In contrast, the use of naked gene therapy, a plasmid expressing the human Maxi-K potassium channel, is the only gene therapy treatment to be evaluated

Histidine metabolism boosts cancer therapy

Methotrexate was one of the first approved anticancer drugs, and is a cornerstone of modern chemotherapy for the treatment of certain solid tumours and blood cancers. However, this therapy must often ... folate in healthy cells — a side effect that is only partially relieved by taking folate supplements. If this toxicity reaches a level that compromises the function of healthy cells, therapy may have to be

Gene Combination Transfer to Block Autoimmune Damage in Transplanted Islets of Langerhans

Islet transplantation therapy would be applicable to a wider range of diabetic patients if donor islet acceptance and protection were possible without systemic immunosuppression of the recipient. To ... being explored to improve islet replacement therapy is to manipulate donor islets before transplantation by ex vivo gene transfer to protect the graft from destruction and circumvent the need for systemic

Advances in Endoscopic Ultrasound, Part 2: Therapy

I study in patients with solid tumors . J Clin Oncol 2004 ; 22 : 592 - 601 . 62. Chang KJ , Senzer N , Chung T , et al. A novel gene transfer therapy against pancreatic cancer (TNFerade) delivered by ... Can J Gastroenterol Advances in endoscopic ultrasound, part 2: Therapy Edward Kim Jennifer J Telford FRCPC - Dr Jennifer J Telford is a gastroenterologist at St Paul’s Hospital and a clinical

Dystrophin Expressing Chimeric (DEC) Human Cells Provide a Potential Therapy for Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a progressive and lethal disease caused by mutations of the dystrophin gene. Currently no cure exists. Stem cell therapies targeting DMD are challenged by limited ... (GM) of dystrophin-deficient mdx/scid mice. This study establishes DEC as a potential therapy for DMD and other types of muscular dystrophies. Duchenne muscular dystrophy; Stem cells; Myoblasts

High-fidelity CRISPR/Cas9- based gene-specific hydroxymethylation rescues gene expression and attenuates renal fibrosis

therapy. TET enzymes catalyze hydroxymethylation of methylated DNA, reactivating gene expression. We here report generation of a high-fidelity CRISPR/Cas9-based gene-specific dioxygenase by fusing an ... previous reports with respect to gene-specific demethylation both in vitro and in vivo, to our knowledge, this study is the first to describe an effective CRISPR-based epigenetic therapy in a disease model

Cholesteryl oleate-loaded cationic solid lipid nanoparticles as carriers for efficient gene-silencing therapy

Cholesteryl oleate-loaded cationic solid lipid nanoparticles as carriers for efficient gene-silencing therapy Marc Suñé-Pou,1–3 Silvia Prieto-Sánchez,2 Younes El Yousfi,2 Sofía Boyero-Corral,2 Anna ... -Roig,1 carlos suñé 2 Introduction The development of efficient delivery systems is a critical factor for successful gene therapy.1 Numerous studies have described the use of vectors, such as

Genome-wide association study of response to tumour necrosis factor inhibitor therapy in rheumatoid arthritis

–6 months. The rs7195994 variant at the FTO gene locus was associated with infliximab response when looking at a change in the swollen joint count (SJC28) subcomponent (p = 9.74 × 10−9). Capture Hi-C ... , Saevarsdottir S, Miceli C, Diogo D, Trynka G, et al. Genome-wide association study and gene expression analysis identifies CD84 as a predictor of response to etanercept therapy in rheumatoid arthritis. Plos Genet

Gene therapy for primary immune deficiencies: a Canadian perspective

The use of gene therapy (GT) for the treatment of primary immune deficiencies (PID) including severe combined immune deficiency (SCID) has progressed significantly in the recent years. In particular ... -myeloablative pretransplantation conditioning in gene therapy for ADA SCID [18–20]. Recently, long-term follow-up (range, 2.3– 13.4 years) of the 18 ADA-deficient patients who received ADA GT in Milan revealed

Differentially expressed genes related to major depressive disorder and antidepressant response: genome-wide gene expression analysis

antidepressant treatment response, we performed gene expression analyses using peripheral blood specimens from 38 MDD patients and 14 healthy individuals at baseline and at 6 weeks after the initiation of either ... . & Tsai, S. J. Genome-wide microarray analysis of gene expression profiling in major depression and antidepressant therapy. Prog. Neuropsychopharmacol. Biol. Psychiatry 64, 334–340 (2016). ArticlePubMed

Oxygen Therapy

pulmonary blood vessels. Oxygen therapy is used to break this cycle. A person with low blood oxygen will often be able to accomplish more with less fatigue with the help of supplemental oxygen therapy ... Can Respir J Oxygen therapy Bonnie Solmes BSW CSW Social work consultant Respiratory Unit West Park Hospital Toronto Ontario The following comments are designed to help patients and their family

Emerging Governance of Mitochondrial Replacement Therapy: Assessing Coherence Between Scientific Evidence and Policy Outcomes

In the fall of 2016, media headlines reported news of several births of children born through “three parent IVF” or mitochondrial replacement therapy (MRT). MRT refers to an experimental procedure ... are prohibited. Aligned with this prohibition, in 2001 the European Union promulgated a directive on clinical trials that further specified, “No gene therapy trials may be carried out which result in

Angiogenic and pleiotropic effects of VEGF165 and HGF combined gene therapy in a rat model of myocardial infarction

Since development of plasmid gene therapy for therapeutic angiogenesis by J. Isner this approach was an attractive option for ischemic diseases affecting large cohorts of patients. However, first

Gene therapy for human glioblastoma using neurotropic JC virus-like particles as a gene delivery vector

cells and oligodendrocytes and causes fatal progressive multifocal leukoencephalopathy in patients with AIDS. In this study, a possible gene therapy strategy for GBM using JCPyV virus-like particles (VLPs ... a therapeutic gene, thus demonstrating the potential of JCPyV VLPs to serve as a gene therapy vector for the far highly treatment-refractory GBM. Introduction Glioblastoma multiforme (GBM) is the

Physical therapy for individuals with amyotrophic lateral sclerosis: current insights

Physical therapy for individuals with amyotrophic lateral sclerosis: current insights Vanina Dal Bello-Haas Physiotherapy Program, School of Rehabilitation Science, McMaster University, Hamilton, ON ... optimal, comprehensive care. In addition to the other health care professions making up the health care team, physical therapy provides a critical role in the overall management in individuals with ALS