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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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Authors: Saito Y, Nakamura K, Ito H Abstract The number of permanent pacemaker implantations has been increasing due to the aging of populations worldwide and the increase in the numbers of patients with heart diseases. Commercially available mechanical pacemakers are very useful but still have some problems including short battery life, a risk of infection, the absence of physiological autonomic responsiveness, metal allergy, and electronic interference. A biological pacemaker may resolve these problems and regenerate the cardiac pacemaker. Cell-based therapy and gene therapy have been addressed with the goal of s...
Source: Acta Medica Okayama - Category: General Medicine Tags: Acta Med Okayama Source Type: research
Publication date: Available online 23 February 2018 Source:Best Practice & Research Clinical Obstetrics & Gynaecology Author(s): Aamod Nawathe, Anna L. David Fetal growth restriction (FGR) and related placental pathologies such as pre-eclampsia and stillbirth arise in early pregnancy when inadequate remodelling of maternal spiral arteries leads to persistent high-resistance low-flow uteroplacental circulation. Current interventions concentrate on targeting the placental ischaemia, re-perfusion injury and oxidative stress associated with an imbalance in angiogenic/anti-angiogenic factors. Recent meta-analyses confi...
Source: Best Practice and Research Clinical Obstetrics and Gynaecology - Category: OBGYN Source Type: research
Human Gene Therapy , Vol. 0, No. 0.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy , Vol. 0, No. 0.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
The present day organization of medical practice and its regulation is built atop the infectious disease model, even where it engages with age-related diseases. Prevention is a comparatively thin thread in an industry largely focused on the strategy of waiting until there is a problem, then attacking the symptoms of that problem with every available tool, as aggressively as possible. This isn't all that useful for age-related disease to start with, but it simply doesn't work for a world in which rejuvenation therapies that can repair the damage that causes aging initially arrive in a prototype form and then grow more capab...
Source: Fight Aging! - Category: Research Authors: Tags: Daily News Source Type: blogs
-Schmutz I, Cavazzana M Abstract Sickle cell disease is characterized by chronic anaemia and vaso-occlusive crises, which eventually lead to multi-organ damage and premature death. Haematopoietic stem cell transplantation is the only curative treatment but it is limited by toxicity and poor availability of HLA-compatible donors. A gene therapy approach based on the autologous transplantation of lentiviral-corrected haematopoietic stem and progenitor cells was shown to be efficacious in one patient. However, alterations of the bone marrow environment and the red blood cells' properties hamper the harvesting and imm...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
The first human trial of gene therapy to treat respiratory problems in early-onset Pompe disease of children was found safe, according to health researchers.
Source: Health News - UPI.com - Category: Consumer Health News Source Type: news
Contributors : Pratiksha I Thakore ; Jennifer B Kwon ; Christopher E Nelson ; Douglas C Rouse ; Matthew P Gemberling ; Matthew L Oliver ; Charles A GersbachSeries Type : Expression profiling by high throughput sequencingOrganism : Mus musculusCRISPR-Cas9 transcriptional repressors have emerged as robust tools for disrupting gene regulation in vitro but have not yet been adapted for delivery in adult animal models. Here we created an S. aureus Cas9-based transcriptional repressor (dSaCas9KRAB) compatible with adeno-associated viral (AAV) delivery. To evaluate dSaCas9KRAB efficacy for targeting an endogenous gene in vivo, we...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Expression profiling by high throughput sequencing Mus musculus Source Type: research
In this study, we used concomitant intratumoral GET of two cytokines: tumor necrosis factor alpha (TNF α), a potent cytotoxic cytokine to induce in situ vaccination, and interleukin 12 (IL-12), an immunostimulatory cytokine to boost the primed local immune response into a systemic one. After performing GET in murine melanoma tumors, both TNFα and IL-12 mRNA levels were significantly increased, whic h resulted in a pronounced delay in tumor growth of 27 days and a prolonged survival time of mice. An antitumor immune response was confirmed by extensive infiltration of immune cells in the tumor site, and expansion...
Source: Cancer Immunology, Immunotherapy - Category: Cancer & Oncology Source Type: research
Voyager will make gene therapies to deliver AbbVie antibodies that target tau, a protein associated with Alzheimer ’s
Source: Chemical and Engineering News - Category: Chemistry Authors: Source Type: research
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