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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

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

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

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

Survival in children requiring chronic renal replacement therapy

<20 years of age in the developed world require renal replacement therapy Improvements in patient survival Since the introduction of the first pediatric chronic RRT programs during the 1960s ... survival probabilities of pediatric renal replacement therapy (RRT) patients by country and period 1 Four-year survival probability 2 Incident dialysis patients only Factors associated with mortality Age

ETS-targeted therapy: can it substitute for MEK inhibitors?

efficacy is still unsatisfactory in the majority of cancers. To substitute ETS-targeted therapy, therapeutic strategies to modulate the transcription factor in cancer must be considered. Chemical targeting ... of Ets transcription factors: interactions, interfaces and inhibition . Biochem Soc Trans 42 : 130 - 138 83. Taniguchi H , Fujiwara Y , Doki Y et al ( 2007 ) Gene therapy using ets-1 transcription

Advancing chimeric antigen receptor T cell therapy with CRISPR/Cas9

chimeric antigen receptor (CAR) T cell-based adoptive immunotherapy. Different methods used to facilitate efficient CRISPR delivery and gene editing in T cells are compared. The potential of genetic ... years, we believe that the CRISPR/ Cas9 technology holds immense promise for advancing immunotherapy. CRISPR/Cas9; chimeric antigen receptor; T lymphocytes; adoptive immunotherapy; gene therapy - The

Intermittent pacing therapy favorably modulates infarct remodeling

Despite early revascularization, remodeling and dysfunction of the left ventricle (LV) after acute myocardial infarction (AMI) remain important therapeutic targets. Intermittent pacing therapy (IPT ... . Intermittent pacing therapy (IPT) of the LV can limit infarct size, when applied during early reperfusion. However, the effects of IPT on post-AMI LV remodeling and infarct healing are unknown. We therefore

Regulation of Cardiomyocyte T-Tubular Structure: Opportunities for Therapy

of heart failure, the plasticity of these structures also creates an opportunity for therapy. Promising recent data suggest that such therapies may specifically target junctophilin-2, BIN1, and/or ... reverse remodeling induced by SERCA2a gene therapy [108]. Workload-dependent regulation of t-tubules has important therapeutic implications. As noted above, this mechanism has already been linked to the

WIP1 phosphatase as pharmacological target in cancer therapy

endangered by erroneous DNA metabolism as well as by various environmental factors that include ionizing radiation or chemotherapy representing two major non-surgical approaches in cancer therapy. Cells ... and its potential use in cancer therapy. DNA damage response and role of WIP1 in checkpoint recovery Various kinds of genotoxic stress activate kinases of PI3kinase like family, including activation of

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

Rapid eradication of colon carcinoma by Clostridium perfringens Enterotoxin suicidal gene therapy

evaluation of a selective and targeted cancer gene therapy of claudin-3- and/or claudin-4- expressing colon carcinoma in vitro and in vivo by using translation optimized CPE expressing vector. Methods In this ... recombinant CPE and a translation optimized CPE expressing vector (optCPE) was used for targeted gene therapy of claudin-3 and/or -4 overexpressing colon cancer cell lines. All experiments were performed in the

Systemic administration of mesenchymal stem cells combined with parathyroid hormone therapy synergistically regenerates multiple rib fractures

been shown to regenerate various tissues. We hypothesized that parathyroid hormone (PTH) therapy would enhance MSC homing and differentiation, ultimately leading to bone formation that would bridge rib ... . examined direct gene therapy via an adenoviral vector and indirect gene therapy in which transduced cells were employed to introduce bone morphogenetic protein (BMP) to the injury site. The results, achieved

Enzyme/Prodrug Systems for Cancer Gene Therapy

significant progress of different suicide gene therapy protocols in preclinical studies and early clinical trials, none has reached the clinic due to several shortcomings. These include slow prodrug-drug ... emphasis on reporting the results of the recent preclinical and clinical studies. Enzyme prodrug; GDEPT; Suicide gene therapy; Bystander effect; Nitroreductase; Cytosine daminase; Thymidine kinase; Carboxyl

Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives

Gene therapy protocols require robust and long-term gene expression. For two decades, retrovirus family vectors have offered several attractive properties as stable gene-delivery vehicles. These ... , accessed in July, 2016). The first successful gene therapy protocol occurred in the 1990s. In that protocol, two patients with severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA

SABCS 2016: systemic therapy for metastatic breast cancer

under the curve BRCA1/2 Breast cancer gene 1 and 2 ctDNA Circulating tumor DNA DNA Deoxyribonucleic acid HER2 Human epidermal growth factor receptor 2 HR Hazard ratio HRD Homologous recombination ... sequentially as mono or combination treatments represent the mainstay of therapy for this disease. At the 2016 San Antonio Breast Cancer Symposium (SABCS) several trials which were designed to overcome drug

A breast cancer gene signature for indolent disease

Purpose Early-stage hormone-receptor positive breast cancer is treated with endocrine therapy and the recommended duration of these treatments has increased over time. While endocrine therapy is ... therapy to aid in personalizing endocrine treatment. Methods We describe a new ultralow risk threshold for the 70-gene signature (MammaPrint) that identifies a group of breast cancer patients with excellent

Fracture Healing Is Delayed in Immunodeficient NOD/scid‑IL2Rγcnull Mice

fracture healing remains poorly understood. Here, we investigated bone healing in NOD/scid-IL2Rγcnull mice, which exhibit severe defects in innate and adaptive immunity, by biomechanical testing

Gene Therapy: A Toolkit for Target Cancer

severe side effects and often prove ineffective at completely eradicating malignant cells. Therefore, a more selective method of targeting tumour cells must be designed. Gene therapy holds great potential ... challenges before they can be used to treat cancer patients on a large scale. Early gene therapy trials such as the SCID-XI trial in 2000 have called into question the safety of gene therapy, particularly the

Increasing the safety and efficacy of chimeric antigen receptor T cell therapy

Chimeric antigen receptor (CAR) T cell therapy is a promising cancer treatment that has recently been undergoing rapid development. However, there are still some major challenges, including precise ... lymphocytes; gene therapy; gene editing - Immunotherapy has become a promising novel cancer treatment and is entering a new era of rapid growth. Different from traditional therapies for cancer patients, such