Advanced search    

Search: Gene Therapy SCID

The page you are looking for is no longer available: Gene Therapy for SCID. Showing search results instead.

504,421 papers found. Showing first 1,000 results.
Use AND, OR, NOT, +word, -word, "long phrase", (parentheses) to fine-tune your search.

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 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

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

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

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

Gene editing in birds takes flight

The application of gene editing (GE) technology to create precise changes to the genome of bird species will provide new and exciting opportunities for the biomedical, agricultural and biotechnology ... species, and conceivably soon other avian species, has joined a growing number of model organisms in the gene editing revolution. - Bird germline transgenesis The chicken has been an exceedingly useful

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

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

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

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

2015 Japanese Society for Dialysis Therapy: Guidelines for Renal Anemia in Chronic Kidney Disease

Renal anemia is a complication of chronic kidney disease. Guidelines for safe and effective treatment in patients with renal anemia are needed. The Japanese Society for Dialysis Therapy (JSDT ... for Dialysis Therapy (JSDT) published guidelines for the treatment of renal anemia in chronic hemodialysis patients in 2004 and in hemodialysis, peritoneal dialysis, predialysis, and pediatric patients

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

Testosterone Therapy on Active Surveillance and Following Definitive Treatment for Prostate Cancer

Purpose of Review Previously considered an absolute contraindication, the use of testosterone therapy in men with prostate cancer has undergone an important paradigm shift. Recent data has changed ... testosterone deficiency in men with prostate cancer. In the current review, we summarize and analyze the literature surrounding effects of testosterone therapy on patients being treated in an active surveillance

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

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

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

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