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Search: Gene Therapy SCID

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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

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

Novel regenerative therapy for acute kidney injury

medicine, however, offers potentially direct AKI therapy through two strategies: cell transplantation and kidney reconstruction. Regarding cell transplantation, several cell types are potential sources ... tissues or secrete paracrine factors that help recovery from the kidney injury. Several cell types have been reported as the source for cell therapy, including hematopoietic stem cells (HSCs), mesenchymal

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

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

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

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

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

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

Suppression of the inflammatory response by disease-inducible interleukin-10 gene therapy in a three-dimensional micromass model of the human synovial membrane

Background Gene therapy has the potential to provide long-term production of therapeutic proteins in the joints of osteoarthritis (OA) patients. The objective of this study was to analyse the ... production of synovial IL-1β and IL-6. Conclusions: Synovial micromasses are a suitable model to test disease-regulated gene therapy approaches and the CXCL10p-IL10 vector might be a good candidate to decrease

Group schema therapy versus group cognitive behavioral therapy for social anxiety disorder with comorbid avoidant personality disorder: study protocol for a randomized controlled trial

patients suffer from both SAD and APD, the Dutch multidisciplinary guidelines for personality disorders advise offering prolonged cognitive behavioral therapy (CBT). Recently there is increasing evidence for ... -group randomized controlled trial (RCT) in which the treatment effect of group cognitive behavioral therapy (GCBT) will be compared to that of group schema therapy (GST) in a semi-open group format. A

Epigenetic polypharmacology: from combination therapy to multitargeted drugs

success of combination therapy, the use of a single drug that modulates several targets might be therapeutically advantageous over the use of drugs in combination. In cancer, the design and synthesis of new ... network perspective [3], to confidently predict new molecular targets for known drugs, and to explain polypharmacology. Another current trend in therapy is drug repurposing or the re-discovery of a new

Treatment of acquired drug resistance in multiple myeloma by combination therapy with XPO1 and topoisomerase II inhibitors

levels found in parental myeloma cell lines. NOD/SCID-γ mice challenged with drug-resistant or parental U266 human MM and treated with selinexor/PLD had significantly decreased tumor growth and increased ... inhibiting apoptosis. Doxorubicin-resistant 8226Dox6 and 8226Dox40 cell lines were produced by the incremental addition of doxorubicin [34]; these cell lines were found to overexpress the MDR1 gene, which

Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world

Gene therapies are classified into two major categories, namely, in vivo and ex vivo. Clinical trials of human gene therapy began with the ex vivo techniques. Based on the initial successes of gene ... world Since the first gene therapy of a monogenic disease, severe combined immunodeficiency due to ADA deficiency (ADA-SCID) was conducted safely and with clinical benefits [ 2 ]; 209 clinical protocols

Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

Background Synthetic zinc finger (ZF) proteins can be targeted to desired DNA sequences and are useful tools for gene therapy. We recently developed a ZF transcription repressor (ZF-KOX1) able to ... single-injection gene therapy approach in the brain. Method: Since non-self proteins can elicit immune and inflammatory responses, we designed a host-matched analogue of ZF-KOX1 (called mZF-KRAB), to treat

Cytotoxic and targeted therapy for hereditary cancers

arising in patients with tuberous sclerosis (TSC), and SMO inhibitors for basal-cell nevus syndrome. Germ-line mutation tests will be increasingly used in the future for the choice of the optimal therapy ... . Hereditary cancer syndromes; Familial cancer; Breast cancer; Ovarian cancer; Colorectal cancer; Cytotoxic therapy; Targeted therapy; Predictive markers; BRCA1; BRCA2 Background First tumor-predisposing germ

Chimeric Antigen Receptors Modified T-Cells for Cancer Therapy

, and discuss the challenges and future prospects for CAR T-cells in cancer therapy. ... thereby Some studies used transcription activator-like effector nuclease enhance the antitumor function of CAR T-cell therapy. However, (TALEN) gene-editing technology to permanently knock out TCR these

Mindfulness-based cognitive therapy for psychological distress in pregnancy: study protocol for a randomized controlled trial

result of receiving mindfulness training in pregnancy. The proposed trial will determine the effectiveness of an 8-week modified Mindfulness-based Cognitive Therapy (MBCT) intervention delivered during ... for further development and testing of interventions to manage psychological distress and anxiety in pregnancy [29, 30]. While there is a small research base using cognitive behavior therapy (CBT) and

Long term disease free survival with multimodal therapy in small cell bladder cancer

recurrence-free. Conclusion In this case report, we were able to show that early multimodal therapy results in long term disease free survival, thus we highly recommend neoadjuvant chemotherapy as a part of ... neoadjuvant chemotherapy as a part of multimodal management of a primary metastases-free, localized and surgically resectable SCBC. Small cell bladder cancer; Multimodal therapy; Neoadjuvant chemotherapy