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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

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

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

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

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

From mechanisms to therapy: RNA processing’s impact on human genetics

: 1345 - 1356 Singh NN , Howell MD , Androphy EJ , Singh RN ( 2017 ) How the discovery of ISS-N1 led to the first medical therapy for spinal muscular atrophy . Gene Ther. doi:10 .1038/gt. 2017 .34 Sterne ... processing solved numerous molecular mechanisms underlying the catalysis of splicing, polyadenylation, RNA editing and translation. Pre-mRNA processing has major impacts on gene expression. The human genome

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

Alcohol consumption and breast tumor gene expression

expressed genes by pre-diagnostic alcohol intake. For pathway analysis, we performed gene set enrichment analysis (GSEA). Differentially expressed genes or enriched pathway-defined gene sets with false ... the NHS/NHSII. However, GSEA identified 33 and 68 pathway-defined gene sets at FDR <0.1 among 471 ER+ and 127 ER- tumors, respectively, all of which were validated. Among ER+ tumors, consuming 10+ grams

mTOR co-targeting strategies for head and neck cancer therapy

; Precision therapy; Immune oncology - Zhiyong Wang and Juan Callejas Valera contributed equally to this work. 1 Background Head and neck squamous cell carcinoma (HNSCC) is the sixth most common malignancy ... % mutated), FAT1 (23% mutated and 5% deleted), NOTCH1 (9% mutated and 66% signaling pathway alterations), CASP8 (10% mutated), CDKN2A (22% mutated and 60% gene copy loss) genes, and PIK3CA (~20% mutated and

Identifying novel genes and biological processes relevant to the development of cancer therapy-induced mucositis: An informative gene network analysis

who were treated using chemotherapy and/or radiation therapy (186 head and neck cancer patients with oral mucositis vs. 699 head and neck cancer patients without oral mucositis). The top ranked gene ... which ERCC1, XRCC1, and MTHFR were the most frequently studied for mucositis. On the basis of this 27-gene list, we used IPA to generate gene networks for mucositis. The most biologically significant

Early assessment of response to induction therapy in acute myeloid leukemia using 18F-FLT PET/CT

Background We evaluated the suitability of 18F-fluorodeoxythymidine (18F-FLT) positron emission tomography (PET)/computed tomography (CT) for assessment of the early response to induction therapy and ... , such as age, cytogenetic risk, and gene mutations at diagnosis. More recently, adjustment of therapy based on incorporation of post-treatment data is likely to become increasingly important. When added

Targeted AAV5-Smad7 gene therapy inhibits corneal scarring in vivo

corneal fibrosis. Further, histopathology revealed lack of immune cell infiltration following AAV5-Smad7 gene transfer into the corneal stroma. Our data demonstrates that AAV5-Smad7 gene therapy is ... need to develop novel therapies for treatment of corneal disorders. Gene therapy is one of the most promising approaches for the treatment of ocular surface disorders, including corneal scarring [6±10

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

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

Risk of gastrointestinal perforation in patients taking oral fluoroquinolone therapy: An analysis of nationally representative cohort

also found that longer durations of fluoroquinolone therapy were associated with a higher incidence of gastrointestinal perforation. The risk increase of gastrointestinal perforation was more substantial ... fluoroquinolone use changed from 15 days to 1±3 months [ 35 ]. Our results, demonstrating a higher risk of gastrointestinal perforation within 60 days of fluoroquinolone therapy, are in concordance with the