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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

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

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

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

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

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

Early changes in gene expression and inflammatory proteins in systemic juvenile idiopathic arthritis patients on canakinumab therapy

potential markers of response using samples from two pivotal trials in systemic juvenile idiopathic arthritis (SJIA). Methods Gene expression was measured in patients with febrile SJIA and in matched healthy ... by baseline expression values. Analysis of gene expression profiles from patients achieving ≥50 aACR JIA response at day 15 identified 102 probe sets differentially expressed upon treatment (≥2-fold

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

Polymeric Nanoparticle-Mediated Gene Delivery for Lung Cancer Treatment

In recent years, researchers have focused on targeted gene therapy for lung cancer, using nanoparticle carriers to overcome the limitations of conventional treatment methods. The main goal of ... methods. The main goal of targeted gene therapy is to develop more efficient therapeutic strategies by improving the bioavailability, stability, and target specificity of gene therapeutics and to reduce off

Case report: rapid and durable response to PDGFR targeted therapy in a child with refractory multiple infantile myofibromatosis and a heterozygous germline mutation of the PDGFRB gene

remains challenging. Patients that harbor an actionable mutation in the kinase domain are potential subjects for targeted tyrosine kinase inhibitor therapy. Case presentation An infant boy with inborn

DNA repair kinetics in SCID mice Sertoli cells and DNA-PKcs-deficient mouse embryonic fibroblasts

-deficient (Prkdc scid ) and wild-type Sertoli cells. In nonirradiated mice and Prkdc scid Sertoli cells displayed persistent DSBs foci in around 12 % of cells and a fivefold increase in numbers of these DSB ... deficiency in NHEJ DNA repair which is associated with early cellular senescence and compromised growth (Ahmed et al. 2013; Gu et al. 1997; Nussenzweig et al. 1997) . In contrast, DNA-PKcsdeficient SCID mice

Intraoperative radiation therapy (IORT) in pancreatic cancer

Despite the important improvements made in the fields of surgery, chemotherapy and radiation therapy, pancreatic cancer remains one of the most lethal malignancies. Improved outcomes with novel ... ) limits the dose of radiation that can be administered to the tumor bed with conventional external beam radiation therapy (EBRT). The intraoperative radiotherapy (IORT) appears to be an ideal therapeutic

Triple therapy versus sequential therapy for the first-line Helicobacter pylori eradication

Background The eradication rate of Helicobacter pylori (H. pylori) with triple therapy which was considered as standard first-line treatment has decreased to 70–85%. The aim of this study is to

Stem cells isolated from human dental pulp and amniotic fluid improve skeletal muscle histopathology in mdx/SCID mice

evaluate their contribution in the improvement of the pathological features associated with dystrophic skeletal muscle when intramuscularly injected into mdx/SCID mice, an immune-compromised animal model of ... cell and gene therapies, have been tested over the years. Cell therapy could be used to recover the lack of dystrophin in dystrophic muscles and thus repair the damaged muscle fibers and prevent future

Calcitonin gene-related peptide and pain: a systematic review

Background Calcitonin gene-related peptide (CGRP) is widely distributed in nociceptive pathways in human peripheral and central nervous system and its receptors are also expressed in pain pathways ... nociceptive processing and therapy of chronic pain. Methods Data extraction The authors (WSS) examined the abstracts found in the literature search. Whenever the title or abstract suggested that relevant data

Neue Hoffnung bei ADA-SCID

Folgeerscheinungen eines Androgen-Überschusses bei Frauen, wie Seborrhoe, fettiges Haar oder Hirsutismus. Nach Informationen von Aristo Pharma Neue Ho nung bei ADA-SCID Ein vollständiger Mangel des Enzyms ... Adenosindesaminase (ADA) führt zum klinischen Bild eines schweren kombinierten Immundefekts (SCID). Bei ADASCID handelt es sich um eine seltene genetische Erkrankung, von der pro Jahr rund 15 Kinder in Europa betro„en

Short-course rapamycin treatment enables engraftment of immunogenic gene-engineered bone marrow under low-dose irradiation to permit long-term immunological tolerance

conditions where both outcomes may be helpful. Hematopoietic stem and progenitor cell (HSPC)-mediated gene therapy often requires highly toxic pretransfer recipient conditioning to provide a ‘niche’ so that ... , Selz F , Hue C , Certain S , Casanova JL , et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease . Science . 2000 ; 288 : 669 - 72 . 2. Cartier N , Hacein-Bey-Abina S