FDA Considers Gene Therapy for Rare Eye Disease

If approved, it would be only the 2nd gene therapy OK'd in the United States
Source: WebMD Health - Category: Consumer Health News Source Type: news

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ConclusionrAAV-GPE-hG6PC treatment in GSD-Ia dogs was found to be safe and efficacious. GSD-Ia is an attractive target for human gene therapy since it is a monogenic disorder with limited tissue involvement. Blood glucose and lactate monitoring can be used to assess effectiveness and as a biomarker of success. GSD-Ia can also serve as a model for other hepatic monogenic disorders.
Source: Journal of Inherited Metabolic Disease - Category: Internal Medicine Source Type: research
MicroRNA21 and the various types of myeloid leukemia, Published online: 25 May 2018; doi:10.1038/s41417-018-0025-2MicroRNA21 and the various types of myeloid leukemia
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
Effect of PAK1 gene silencing on proliferation and apoptosis in hepatocellular carcinoma cell lines MHCC97-H and HepG2 and cells in xenograft tumor, Published online: 25 May 2018; doi:10.1038/s41434-018-0016-9Effect of PAK1 gene silencing on proliferation and apoptosis in hepatocellular carcinoma cell lines MHCC97-H and HepG2 and cells in xenograft tumor
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Authors: Kenda Suster N, Virant-Klun I, Frkovic Grazio S, Smrkolj S Abstract Ovarian cancer is among the most common gynecologic cancers and unfortunately the most common cause of death from gynecologic malignancies. Due to few early symptoms and insufficient screening programs, an early diagnosis of ovarian cancer is very difficult and new biomarkers related to early ovarian carcinogenesis are needed. In the last years a growing scientific knowledge about cancer stem cells and their markers opened a new perspective on screening and early diagnosis of ovarian cancer. The transcription factor NANOG is not only a plu...
Source: European Journal of Gynaecological Oncology - Category: OBGYN Tags: Eur J Gynaecol Oncol Source Type: research
Sickle cell disease is the most prevalent monogenic disorder worldwide and curative therapies are limited to hematopoietic stem cell transplant to the few with matched donors. Gene therapy has curative potential, whereby autologous hematopoietic stem cells are genetically modified and transplanted, which would not be limited by matched donors, resulting in 1-time, life-long correction devoid of immune side effects. Significant progress has been made to clinically translate gene therapy for sickle cell disease using lentivirus vectors carrying antisickling genes. This review focuses on the current state of the field, factor...
Source: Pediatric Clinics of North America - Category: Pediatrics Authors: Source Type: research
This article focuses on supportive and preventive care improvements and the benefits of hydroxyurea. Indications for erythrocyte transfusion, hematopoietic stem cell transplant, and gene therapy trials are also summarized.
Source: Pediatric Clinics of North America - Category: Pediatrics Authors: Source Type: research
Rapid expansion of therapeutic options have increased the complexity of hemophilia care. Previously, on-demand therapy aimed to reduce morbidity and early mortality; however, now aggressive prophylaxis, particularly in children, encourages an active lifestyle. Accurate diagnosis, recognition of early threats to musculoskeletal health, and optimization of therapy are critical for both males and females affected by hemophilia. The diversity of emerging hemophilia therapies, from modified factor protein concentrates, to gene therapy, to nonfactor hemostatic strategies, provide an exciting opportunity to target unmet needs in ...
Source: Pediatric Clinics of North America - Category: Pediatrics Authors: Source Type: research
Specific inhibitor of Notch‑3 enhances the sensitivity of NSCLC cells to gemcitabine. Oncol Rep. 2018 May 16;: Authors: Hu BD, Guo J, Ye YZ, Du T, Cheng CS, Jiang Q, Liu RN, Zhang YB Abstract Notch‑3 is a receptor of the Notch signaling pathway and plays an important role in regulating self‑renewal, differentiation and apoptosis in cancer cells. Overexpression of Notch‑3 has been proved to be associated with resistance to gemcitabine (GEM) and poor patient prognosis for various malignant tumors. In the present study, two non‑small cell lung cancer (NSCLC) cell lines, H1299 and A549, were indu...
Source: Oncology Reports - Category: Cancer & Oncology Tags: Oncol Rep Source Type: research
Publication date: Available online 19 May 2018 Source:Journal of Molecular Biology Author(s): Anne-Kathrin Herrmann, Dirk Grimm Over fifty years after its initial description, Adeno-associated virus (AAV) remains a most exciting but also most elusive study object in basic or applied virology. On the one hand, its simple structure not only facilitates investigations into virus biology, but combined with the availability of numerous natural AAV variants with distinct infection efficiency and specificity also makes AAV a preferred substrate for engineering of gene delivery vectors. On the other hand, it is striking to witnes...
Source: Journal of Molecular Biology - Category: Molecular Biology Source Type: research
Adenovirus-mediated transfer of HPV 16 E6/E7 antisense RNA combined with cisplatin inhibits cellular growth and induces apoptosis in HPV-positive head and neck cancer cells, Published online: 24 May 2018; doi:10.1038/s41417-018-0024-3Adenovirus-mediated transfer of HPV 16 E6/E7 antisense RNA combined with cisplatin inhibits cellular growth and induces apoptosis in HPV-positive head and neck cancer cells
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
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