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Sustained intrathecal therapeutic protein delivery using genetically transduced tissue implants in a freely moving rat model

Publication date: 20 December 2017 Source:International Journal of Pharmaceutics, Volume 534, Issues 1–2 Author(s): J.P. Aronson, H.A. Katnani, I. Pomerantseva, N. Shapir, H. Tse, R. Miari, H. Goltsman, O. Mwizerwa, C.M. Neville, G.A. Neil, E.N. Eskandar, C.A. Sundback Systemic delivery of therapeutic proteins to the central nervous system (CNS) is challenging because of the blood-brain barrier restrictions. Direct intrathecal delivery is possible but does not produce stable concentrations. We are proposing an alternative approach for localized delivery into the CNS based on the Transduced Autologous Restorative Gene Therapy (TARGT) system. This system was previously developed using a gene therapy approach with dermal tissue implants. Lewis rat dermal tissue was transduced to secrete human EPO (hEPO). TARGT viability and function were retained following cryopreservation. Upon implantation into the rat cisterna magna, a mild inflammatory response was observed at the TARGT-brain interface throughout 21-day implantation. hEPO expression was verified immunohistochemically and by secreted levels in cerebrospinal fluid (CSF), serum, and in vitro post explant. Detectable CSF hEPO levels were maintained during the study. Serum hEPO levels were similar to rat and human basal serum levels. In vitro, the highest hEPO concentration was observed on day 1 post-explant culture and then remained constant for over 21days. Prolonged incubation within the cisterna magna had no negative i...
Source: International Journal of Pharmaceutics - Category: Drugs & Pharmacology Source Type: research

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A gene therapy startup that spun out of Nationwide Children's Hospital six months ago has raised $2.5 million toward testing a treatment for a form of muscular dystrophy in patients. Myonexus Therapeutics Inc. is the fourth company to come out of gene therapy research at the Columbus hospital and the only one located in Central Ohio. CEO Michael Triplett and COO Bruce Halpryn are based in New Albany, and all of the contract research it sponsors takes place at Children's. “ The clinical impact…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
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Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
Publication date: Available online 12 December 2017 Source:Stem Cell Research Author(s): Yanlin Wang, Na Jing, Linlin Su, Changhe Shi, Pei Zhang, Zhilei Wang, Huifang Sun, Jing Yang, Yutao Liu, Xuejun Wen, Jin Zhang, Shoutao Zhang, Yuming Xu Skin fibroblasts were obtained from a 42-year-old Alzheimer's disease (AD) patient carrying mutations in the PSEN1 gene. An iPSC line was successfully established using the Sendai-virus (SeV) delivery system. The patient-specific iPSCs were free of genomically integrated reprogramming genes, had the specific mutation, expressed the expected pluripotency markers, and had the potential ...
Source: Stem Cell Research - Category: Stem Cells Source Type: research
In conclusion, these findings suggest that interference with PLCγ2 expression could relieve the inhibitory effect of PLCγ2 on hepaocyte apoptosis, thus promote proliferation through inactivating PKCδÔÇÉmediated JNK1/2 signaling pathway. This article is protected by copyright. All rights reserved
Source: Journal of Cellular Biochemistry - Category: Biochemistry Authors: Tags: Article Source Type: research
Condition:   Hemophilia B Intervention:   Biological: FLT180a Sponsor:   University College, London Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
PARIS, December 12, 2017 -- (Healthcare Sales &Marketing Network) -- Eyevensys, a clinical stage biotech company developing non-viral gene therapies for ophthalmic diseases, today announced the appointment of Dr. Patricia Zilliox as CEO. Dr. Zilliox, ... Biopharmaceuticals, Ophthalmology, Personnel Eyevensys, EyeCET, gene therapy, electro-transfection injection
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
LncRNA SPRY4-IT was concerned with the poor prognosis and contributed to the progression of thyroid cancer, Published online: 12 December 2017; doi:10.1038/s41417-017-0003-0LncRNA SPRY4-IT was concerned with the poor prognosis and contributed to the progression of thyroid cancer
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
LncRNA-SNHG16 predicts poor prognosis and promotes tumor proliferation through epigenetically silencing p21 in bladder cancer, Published online: 12 December 2017; doi:10.1038/s41417-017-0006-xLncRNA-SNHG16 predicts poor prognosis and promotes tumor proliferation through epigenetically silencing p21 in bladder cancer
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
Netrin-1 elicits metastatic potential of non-small cell lung carcinoma cell by enhancing cell invasion, migration and vasculogenic mimicry via EMT induction, Published online: 12 December 2017; doi:10.1038/s41417-017-0008-8Netrin-1 elicits metastatic potential of non-small cell lung carcinoma cell by enhancing cell invasion, migration and vasculogenic mimicry via EMT induction
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
by Sudhir Kumar Rai, Maya Sangesland, Michael Lee Jr., Caroline Esnault, Yujin Cui, Atreyi Ghatak Chatterjee, Henry L. Levin Retroviruses and Long Terminal Repeat (LTR)-retrotransposons have distinct patterns of integration sites. The oncogenic potential of retrovirus-based vectors used in gene therapy is dependent on the selection of integration sites associated with promoters. The LTR-retrotransposon Tf1 ofSchizosaccharomyces pombe is studied as a model for oncogenic retroviruses because it integrates into the promoters of stress response genes. Although integrases (INs) encoded by retroviruses and LTR-retrotransposons ...
Source: PLoS Genetics - Category: Genetics & Stem Cells Authors: Source Type: research
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