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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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Voyager Therapeutics said Tuesday that it will work with AbbVie to develop gene therapy treatments for Alzheimer ’s disease in a deal worth at least $69 million for the Cambridge biotech. The announcement by Voyager (Nasdaq: VYGR) and Chicago-based AbbVie (NYSE: ABBV) comes as drug developers are exploring new approaches to Alzheimer’s following a string of recent clinical setbacks. Gene therapies — dru gs that work by inserting healthy genes into a person’s cells to replace faulty or missing…
Source: bizjournals.com Health Care News Headlines - Category: Health Management Authors: Source Type: news
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Source: IEEE Transactions on Biomedical Engineering - Category: Biomedical Engineering Source Type: research
ConclusionBy extrapolating our murine data, and data from some previous studies to a human non‐conditioned autologous CD34+ HSPC transplantation setting, we conclude that approximately 44 million CD34+ HSPCs would be needed to achieve 20% donor chimerism in a 70‐kg human, which could serve as a starting point for the future use of HSCPs in gene and cell therapy.
Source: European Journal of Haematology - Category: Hematology Authors: Tags: ORIGINAL ARTICLE Source Type: research
Gene therapies involve delivering instructions into cells to ensure that specific proteins are manufactured, either temporarily or permanently. This is effectively a hijacking or programming of cellular mechanisms. There is another approach, which is to deliver suitable DNA machinery into the body, capable of manufacturing the desired proteins outside cells. This isn't helpful for all types of protein, but in many cases it is. That machinery needs protection, however: naked, it would be quickly removed by the immune system or otherwise broken down. One possibility is to employ engineered bacteria, which removes the need to...
Source: Fight Aging! - Category: Research Authors: Tags: Daily News Source Type: blogs
Fight Aging! provides a weekly digest of news and commentary for thousands of subscribers interested in the latest longevity science: progress towards the medical control of aging in order to prevent age-related frailty, suffering, and disease, as well as improvements in the present understanding of what works and what doesn't work when it comes to extending healthy life. Expect to see summaries of recent advances in medical research, news from the scientific community, advocacy and fundraising initiatives to help speed work on the repair and reversal of aging, links to online resources, and much more. This content is...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Although recent advances in gene therapy are expected to increase the chance of disease cure in thalassemia major, at present hematopoietic stem cell transplantation (HSCT) remains the only consolidated curative approach for this disorder. The widest experience has been obtained in the HLA-matched family donor (MFD) setting, with probabilities of overall and thalassemia-free survival exceeding 90% and 85%, respectively. As for most patients a suitable MFD is not available, alternative donors (HLA-matched unrelated donor, unrelated cord blood, HLA-haploidentical relative) have been increasingly explored, translating into th...
Source: Hematology/Oncology Clinics of North America - Category: Cancer & Oncology Authors: Source Type: research
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Source: PLOS Currents Muscular Dystrophy - Category: Neurology Authors: Source Type: research
(Mary Ann Liebert, Inc./Genetic Engineering News) China is helping to advance gene and cell therapy and genome editing research and clinical development by creating novel viral and nonviral vectors for gene delivery and innovative applications of CRISPR technology in a broad range of disease areas.
Source: EurekAlert! - Biology - Category: Biology Source Type: news
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Source: European Cells and Materials - Category: Cytology Tags: Eur Cell Mater Source Type: research
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Source: Cellular Physiology and Biochemistry - Category: Cytology Source Type: research
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