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Advances in ovarian cancer therapy

under development, bring the promise of turning ovarian cancer into a manageable chronic disease. In this review, we discuss the current standard in the therapy for ovarian cancer, major recent studies on ... discuss cost-effectiveness of some novel therapies and the issue of better selection of patients for personalized treatment. Ovarian cancer; Biological drugs; Targeted therapy; Clinical trials

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

Pre-operative Endocrine Therapy

Purpose of Review Pre-operative endocrine therapy can be used to down-stage large or locally advanced breast cancers in ER+ disease. In the last four decades, it has evolved from a treatment ... to neoadjuvant therapy has led to exploration of other biomarkers, including Ki67, to predict response to treatment and estimate prognosis. One study assessed 21-gene recurrence score (RS; Oncotype DX

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

Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy

neurological decline in Canavan disease—a fatal neurometabolic disorder caused by deficiency in the NAA-degrading enzyme aspartoacylase. To date clinical outcome of gene replacement therapy for this spongiform ... these findings we executed an ASPA gene replacement therapy targeting oligodendrocytes in Canavan disease mice resulting in reversal of pre-existing Vol.:(011233456789) - CNS pathology and lasting

Gene Editing in Clinical Practice

assisted in gene therapy using induced pluripotent stem cells [ 1 ]. Amongst some genetic tools, the most talked about gene editing tool is CASPR-Cas9 which has made the headlines all over the world ... organism. Here by means of engineered nucleases or molecular scissors the defective DNA is inserted, deleted or replaced. The advent of gene editing technique has generated more excitement in biology and

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 ... provide an overview of the most recent scientific advances in HSCbased gene therapy approaches for the treatment of LSDs with particular focus on metachromatic leukodystrophy (MLD) and mucopolysaccharidosis

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

Positioning of proteasome inhibitors in therapy of solid malignancies

( 2017 ) Combined bortezomib-based chemotherapy and p53 gene therapy using hollow mesoporous silica nanospheres for p53 mutant non-small cell lung cancer treatment . Biomater Sci 5 : 77 - 88 . https ... :// 96. Huang C , Yokomise H , Miyatake A ( 2007 ) Clinical significance of the p53 pathway and associated gene therapy in nonsmall cell lung cancers . Future Oncol 3 : 83 - 93

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

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

Weighted functional linear regression models for gene-based association analysis

Functional linear regression models are effectively used in gene-based association analysis of complex traits. These models combine information about individual genetic variants, taking into account ... combined, weights are introduced to give the advantage to more informative components. Allele-specific weights have been introduced to collapsing and kernel-based approaches to gene-based association

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

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

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

Increased Engraftment of Human Short Term Repopulating Hematopoietic Cells in NOD/SCID/IL2rγnull Mice by Lentiviral Expression of NUP98-HOXA10HD

Techniques to expand human hematopoietic stem cells ex-vivo could be beneficial to the fields of clinical hematopoietic stem cell transplantation and gene therapy targeted at hematopoietic stem cells ... sustain or improve the HSC content in grafts maintained in culture and potentially improve the transfer efficiency in gene therapy where HSCs are the main target. Results Lentiviral vector production The

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