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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

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

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

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

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

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

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

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

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

Metformin – a Future Therapy for Neurodegenerative Diseases

(20), which, in turn, indicates that therapy with metformin may promote the development of AD (21). On the basis of the presented results, and taking into account that T2DM increases the overall risk ... (ApoE) ε4 allele carriers (28). ApoE, a cholesterol carrier in the brain and mediator of the uptake of lipoprotein, is coded by a gene which has three polymorphic alleles: ε2, ε3 and ε4 (34). Among them

Evidence-Based Design of Fixed-Dose Combinations: Principles and Application to Pediatric Anti-Tuberculosis Therapy

multi-drug therapy [ 1, 2 ]. Fixed-dose combinations can apart from reducing the pill burden, also simplify the prescription procedure and the distribution chain in the healthcare system [3]. It is also ... original procedure. For isoniazid, the known global variability in the distribution of arylamine N-acetyltransferase 2 gene variations, which strongly impacts isoniazid clearance, is a complicating factor

Metformin therapy for the reproductive and metabolic consequences of polycystic ovary syndrome

considerable heterogeneity in the clinical response to this therapy in women with PCOS. Recent evidence indicates that genetic factors may play a significant role in predicting response to metformin therapy in

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

Tumor-Associated Macrophages as Target for Antitumor Therapy

immunosuppression (Qian and Pollard 2010; Biragyn and Longo 2012) . Since TAMs influence various aspects of cancer progression, they can serve as a target for clinical therapy. Moreover, accumulating studies have ... therapy. Macrophage-Associated Therapies The regular treatment for cancer patients is aggressive surgery of tumor tissue supported with radiochemical or hormonal therapy. However, none of these treatments

Alzheimer’s disease, dementia, and stem cell therapy

learning and memory, and so promoting this process may help counter the amnestic symptoms of early AD. One option has been to upregulate (pharmacologically or with gene therapy) those growth factors known ... beyond age including cardiovascular disease, low education, depression, and the apolipoprotein-E4 (ApoE4) gene. Sporadic AD is accordingly of multifactorial origins, driven in part by a complex 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

Regression of Neonatal Cardiac Rhabdomyoma in Two Months Through Low-Dose Everolimus Therapy: A Report of Three Cases

-1 and TSC-2 gene abnormality were also negative. Discussion In this cases series of three neonates with CR, we deliberately used a low dose regimen of everolimus therapy, beginning with 0.3–0.67 mg ... providing an explanation to the parents, we began an off-label use of oral low-dose everolimus therapy (0.0625 mg/day, 1/4 of a 0.25 mg tablet, equivalent to 0.334 mg/M2/day), aiming to achieve a trough serum

Outcomes of renal replacement therapy in boys with prune belly syndrome: findings from the ESPN/ERA-EDTA Registry

started renal replacement therapy (RRT) between 1990 and 2013 in 35 European countries. Patient characteristics, survival, and transplantation outcomes were compared with those of male patients requiring ... Renal Association-European Dialysis and Transplant Association (ESPN/ERA-EDTA) Registry data. Methods Data were available for 88 male PBS patients aged <20 years who started renal replacement therapy (RRT