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Tis21-gene therapy inhibits medulloblastoma growth in a murine allograft model

. Overall the results suggest that Tis21-gene therapy slows down MB tumor growth through inhibition of proliferation and enhancement of neural differentiation. These results validate Tis21 as a relevant ... pediatric mortality related to cancer. MB is also www.cnr.it/progetti/ProgettoNuovo.html?id_prog= 9162). FT was also funded by the CNR project DSB.AD004.094 (Medulloblastoma gene therapy; web site. http

Striated muscle gene therapy for the treatment of lipoprotein lipase deficiency

Excessive circulating triglycerides due to reduction or loss of lipoprotein lipase activity contribute to hypertriglyceridemia and increased risk for pancreatitis. The only gene therapy treatment for ... , in a small number of patients by LPL gene therapy using alipogene tiparvovec (S447X variant). Despite tiparvovec reducing the frequency of acute pancreatitis [14,15], it was recently announced that

NK cell therapy for hematologic malignancies

sensitization to induce cytotoxicity and do not cause graft-versus-host disease. These, along with other advantages, make NK cells an attractive candidate for adoptive cellular therapy. Herein, we describe the ... natural killer cells is improved using IL-2 diphtheria toxin fusion protein . Blood . 2014 ; 123 ( 25 ): 3855 - 63 . 112. FDA. FDA approval brings first gene therapy to the United States . https

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

Liver cell therapy: is this the end of the beginning?

chain gene. The knockout creates a lack of functional receptors for many cytokines leading to an impaired lymphocyte development and a lack of NK cells [ 63 ]. Non-obese diabetic (NOD) SCID gamma mice ... deficiency) [15]. Other potential candidates for liver cell therapy include familial hypercholesterolemia and Wilson’s disease [ 16 ] and advanced liver cirrhosis [ 17 ]. In addition to disease-causing

Advances in ovarian cancer therapy

under development, bring the promise of turning ovarian cancer into a manageable chronic disease. In this review, we discuss the current standard in the therapy for ovarian cancer, major recent studies on ... discuss cost-effectiveness of some novel therapies and the issue of better selection of patients for personalized treatment. Ovarian cancer; Biological drugs; Targeted therapy; Clinical trials

Oral mucosa tissue gene expression profiling before, during, and after radiation therapy for tonsil squamous cell carcinoma

Background Radiation-therapy (RT) induces mucositis, a clinically challenging condition with limited prophylactic interventions and no predictive tests. In this pilot study, we applied global gene ... , Denmark , 6 School of Dentistry, Faculty of Health Sciences, Aarhus University; Aarhus , Denmark Radiation-therapy (RT) induces mucositis, a clinically challenging condition with limited prophylactic

Pre-operative Endocrine Therapy

Purpose of Review Pre-operative endocrine therapy can be used to down-stage large or locally advanced breast cancers in ER+ disease. In the last four decades, it has evolved from a treatment ... to neoadjuvant therapy has led to exploration of other biomarkers, including Ki67, to predict response to treatment and estimate prognosis. One study assessed 21-gene recurrence score (RS; Oncotype DX

Doxorubicin-provoked increase of mitotic activity and concomitant drain of G0-pool in therapy-resistant BE(2)-C neuroblastoma

). Considering the clinical challenge for treating high-risk NB, the discovery of a therapy-provoked growth-stimulating effect in the multi-resistant and p53-mutated BE(2)-C cell line, but not in the drug ... metastatic phenotypes [ 2 ]. Metastasis and relapse are the main causes of death, which make it imperative to understand metastatic dissemination and intra-tumour variations for therapy responsiveness. An

Antibody-Conjugated Silica-Modified Gold Nanorods for the Diagnosis and Photo-Thermal Therapy of

evaluated to confirm the photo-thermal therapy effect of GNR-SiO2-Ab combined with near-infrared (NIR) laser light. Results: GNR-SiO2-Ab has a potential application as a positive X-ray/CT imaging contrast ... -induced photo-thermal therapy in C. neoformans cells. Gold nanorods; Cryptococcus neoformans; Diagnosis; Photo-thermal therapy Background Cryptococcus neoformans is an encapsulated yeast, which was first

Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy

neurological decline in Canavan disease—a fatal neurometabolic disorder caused by deficiency in the NAA-degrading enzyme aspartoacylase. To date clinical outcome of gene replacement therapy for this spongiform ... these findings we executed an ASPA gene replacement therapy targeting oligodendrocytes in Canavan disease mice resulting in reversal of pre-existing Vol.:(011233456789) - CNS pathology and lasting

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

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 ... provide an overview of the most recent scientific advances in HSCbased gene therapy approaches for the treatment of LSDs with particular focus on metachromatic leukodystrophy (MLD) and mucopolysaccharidosis

Cancer chemoprevention and therapy using chinese herbal medicine

slightly reduced the incidence of esophageal SCC from 34.5% (10/ 29) to 22.2% (6/27) [ 33 ]. In B[a]P-induced mouse lung adenomas, ATB reverted 40% of gene expression changes to normal levels [ 31 ], and ... via the ERK1/2 signaling pathway as well as MMP-9 activity [ 51 ]. In addition, PV induced gene expression and the production of macrophage-related cytokines, such as TNF-α, IL-1b, and IL-6. PV

Cognitive Behavior Therapy for Fear of Cancer Recurrence: A Case Study

This case study describes the course and content of cognitive behavior therapy (CBT) for clinical fear of cancer recurrence (FCR) in a breast cancer survivor. The CBT for clinical FCR consisted of ... the breast cancer survivor pre-therapy, post-therapy, and at 6 and 12 months of post-therapy. In each treatment session, perceived control over FCR was assessed. A clinical nurse specialist participated

Gene Editing in Clinical Practice

assisted in gene therapy using induced pluripotent stem cells [ 1 ]. Amongst some genetic tools, the most talked about gene editing tool is CASPR-Cas9 which has made the headlines all over the world ... organism. Here by means of engineered nucleases or molecular scissors the defective DNA is inserted, deleted or replaced. The advent of gene editing technique has generated more excitement in biology and

Transcriptomic analysis of gene expression in mice treated with troxerutin

-inflammation and anti-aging. However, the effects of troxerutin on genome-wide transcription in blood cells are still unknown. In order to find out effects of troxerutin on gene transcription, a high-throughput ... , a high-throughput RNA sequencing was employed to analysis differential gene expression in blood cells consisting of leucocytes, erythrocytes and platelets isolated from the mice received subcutaneous

Combination therapy for cancer with oncolytic virus and checkpoint inhibitor: A mathematical model

this paper we consider a combination therapy with OV and a checkpoint inhibitor, anti-PD-1. We evaluate the efficacy of the combination therapy in terms of the tumor volume at some later time, for ... . Cancer Gene Ther . 2009 ; 18 ( 3 ): 167 ± 175 . https://doi.org/10.1038/ cgt. 2010 .65 13. Patel MR , Kratzke RA . Oncolytic virus therapy for cancer: the first wave of translational clinical trials