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Liver cell therapy: is this the end of the beginning?

chain gene. The knockout creates a lack of functional receptors for many cytokines leading to an impaired lymphocyte development and a lack of NK cells [ 63 ]. Non-obese diabetic (NOD) SCID gamma mice ... deficiency) [15]. Other potential candidates for liver cell therapy include familial hypercholesterolemia and Wilson’s disease [ 16 ] and advanced liver cirrhosis [ 17 ]. In addition to disease-causing

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

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV ... patient (Bryant et al 2013). GlaxoSmithKline‘s Strimvelis® is the second gene therapy product to reach the market and was approved by the EMA in 2016. This gene therapy product, which targets ADA-SCID has

Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I

treatment occurs before disease onset. In the attempt to overcome these issues, gene therapy has been proposed as a valuable therapeutic option, either ex vivo, with target cells genetically modified in vitro

Gene Editing, Gene Therapy, and Cell Xenotransplantation: Cell Transplantation Across Species

xenotransplantation although it might be of greater relevance for other tissues. Neural Xenotransplantation and Gene Therapy Neural cell xenotransplantation is an attractive alternative therapy for neurodegenerative ... after transplantation in rats [71]. Genetic modifications can also enhance transplanted cells’ function or even confer new functions. In a primate model of PD, gene therapy by lentiviral expression of

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus ... factors to consider in the use of AAV as a vector for gene therapy. 1 Introduction The discovery of DNA as the biomolecule of genetic inheritance and disease opened up the prospect of therapies in which

Functional evaluation of therapeutic response of HCC827 lung cancer to bevacizumab and erlotinib targeted therapy using dynamic contrast-enhanced and diffusion-weighted MRI

tumor growth, metastasis and patient prognosis [2±4]. Targeted therapy against angiogenesis can lead to regression or normalization of neovascular structures, and the inhibition of new blood vessel growth ... for the treatment of patients with NSCLC are needed [ 8 ]. Somatic mutations in the EGFR gene have been identified in patients who respond to EGFR-tyrosine kinase inhibitors (TKIs) [9±11]. The two

Using a novel cellular platform to optimize CRISPR/CAS9 technology for the gene therapy of AIDS

the CAG promoter into the promoter of the CCR5 gene via homologous recombination. The resulting cell line is permissive for HIV infection and is useful for developing gene therapy of AIDs. We used ... support future HIV research. To further demonstrate the feasibility to use Jurkat-KI-R5 cells for HIV research, we employed the Jurkat-KI-R5 cells to develop gene therapy for AIDS. The highly pursued

Follicular growth after xenotransplantation of cryopreserved/thawed human ovarian tissue in SCID mice: dynamics and molecular aspects

tissue obtained from female cancer patients (n = 11) were xenotransplanted into a subcutaneous neck pouch of 6-week-old ovarectomized SCID mice (n = 33) for 4 (n = 18) and 12 (n = 15) weeks. Result Thirty ... gene expression followed by acceleration of follicular recruitment. - Department of Gynecological Endocrinology and Reproductive Medicine, Medical University of Innsbruck, Anichstr. 35, 6020 Innsbruck

The effects of progesterones on blood lipids in hormone replacement therapy

purpose of this review is to describe the role of varied progestogens in hormone replacement therapy (HRT), especially focusing on blood lipids, which are the most important parameters for assessing ... replacement therapy; Progestogens; Lipids Background There are several diverse psychological symptoms due to estrogen deficiency during menopause including hot flushes, bouts of sweating, poor memory

Tamoxifen therapy benefit for patients with 70-gene signature high and low risk

nil), versus no adjuvant treatment, provides a unique opportunity to evaluate long-term 20-year benefit of endocrine therapy within prognostic risk classes of the 70-gene prognosis signature that was ... randomization for an additional 3-year tamoxifen vs nil), versus no adjuvant treatment, 70-gene signature; Tamoxifen benefit; Endocrine therapy; Breast cancer; Long-term survival - provides a unique

Erratum to: Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease

responsibility of the authors and does not necessarily represent the official views of HRSA. The images in the gene therapy brochure are not pictures of patients but stock photos we obtained through a paid vendor

Bacterial magnetosomes as an efficient gene delivery platform for cancer theranostics

Gene therapy has gained an increasing interest in its anti-tumor efficiency. However, numerous efforts are required to promote them to clinics. In this study, a novel and efficient delivery platform ... successfully produced for cancer therapy, and these innovative carriers will potentially find widespread applications in the pharmaceutical field. Bacterial magnetosomes; siRNA; Polyethylenimine; Gene therapy

Survivin a radiogenetic promoter for glioblastoma viral gene therapy independently from CArG motifs

response to radiation had the lowest number of CCAAT box. Conclusion Survivin is a selective potent radiation inducible promoter for glioblastoma viral gene therapy and this response to radiation could be ... expression regulated by Egr-1 regulated element on radiation injury of SCID mice . Exp Hematol 31 ( 3 ): 191 - 196 30. Schmidt M et al ( 2004 ) Inducible promoters for gene therapy of head and neck cancer: an

Toxoplasma gondii serine-protease inhibitor-1: A new adjuvant candidate for asthma therapy

. Allergen-specific immunotherapy has emerged as an alternative treatment. Still, at present, this kind of therapy presents several concerns related to the type of vaccine and adjuvant used, route of ... , Duschak VG , Paulino M , Ledesma B , Yabo MI , et al. Molecular cloning, sequencing and expression of a serine proteinase inhibitor gene from Toxoplasma gondii . Mol Biochem Parasitol . 2000 ; 107 : 241 ±9

Autologous adipose-derived regenerative cell therapy modulates development of hypertrophic scarring in a red Duroc porcine model

swine, which do not develop HTS [33]. Furthermore, intralesional injection of ADSC in a rabbit ear model reduced α-SMA and collagen type I gene expression at day 35 post-treatment [ 34 ]. Similarly ... extremes of age or compromise. Second, this study assessed only a single dose of the cell therapy product. It may be that other dose regimens will prove more or less effective. Additional investigation will

The Efficacy of Biologic Therapy for the Management of Palmoplantar Psoriasis and Palmoplantar Pustulosis: A Systematic Review

) and PPP. The aim of this review was to investigate the efficacy of biologic therapy for the treatment of hyperkeratotic PP, pustular PP, and PPP. Methods A systematic search of the medical electronic ... electronic databases (Medline, Embase, and Cochrane Library) was conducted to identify studies or case reports which both used biologic therapy for the treatment of hyperkeratotic PP, pustular PP, and PPP and

The Potential of microRNAs for Stem Cell-based Therapy for Degenerative Skeletal Diseases

combination with stem cell therapy could induce skeletal regeneration and potentially prevent OA and OP onset. Recent Findings The combination of stem cells and miRNA has been successful at regenerating the ... the onset of OA and OP remain a significant unmet clinical need. MicroRNAs (miRNAs) are known to be involved in the differentiation of stem cells, and in combination with stem cell therapy could induce

Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma

-derived GBM. AAVrh10.CetMab-treated mice displayed a reduction in cachexia, a significant decrease in tumor volume and a prolonged survival following therapy. Adeno-associated-directed delivery of a gene ... time of tumor implantation and treatment. Gene Therapy for Glioblastoma The challenge for anti-GBM gene therapy is to develop a safe platform that delivers an efficacious dose of an anti-tumor genetic