Therapeutic gene transfer holds the promise of providing lasting therapies and even cures for diseases that were previously untreatable or for which only temporary or suboptimal treatments were available. For some time, clinical gene therapy was characterized by some impressive but rare examples of successes and also several setbacks. However, effective and long-lasting treatments are now being reported from gene therapy trials at an increasing pace. Positive outcomes have been documented for a wide range of genetic diseases (including hematological, immunological, ocular, and neurodegenerative and metabolic disorders) and several types of cancer. Examples include restoration of vision in blind patients, eradication of blood cancers for which all other treatments had failed, correction of hemoglobinopathies and coagulation factor deficiencies, and restoration of the immune system in children born with primary immune deficiency. To date, about 2,000 clinical trials for various diseases have occurred or are in progress, and many more are in the pipeline. Multiple clinical studies reported successful treatments of pediatric patients. Design of gene therapy vectors and their clinical development are advancing rapidly. This article reviews some of the major successes in clinical gene therapy of recent years.
In 2000, measles was declared eliminated in the US. But last year saw a record number of measles cases since - 644 over 27 US states. This year is unlikely to be any better, with 102 cases reported so far.
Pneumonia, gonorrhoea and urinary tract infections that no longer respond to antibiotics are fast becoming a reality worldwide. Last week, global leaders pledged to unite to protect the medicines that protect us.
Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells.
Here’s good news for anyone affected by chikungunya this year: the virus is unlikely to infect them ever again. This has been confirmed to TOI by virologists from AIIMS who said that the chikungunya virus has only one serotype and hence the risk of repeated infection is minimal.
An Asian elephant at the Oklahoma City Zoo has tested positive for the EEHV1A virus for the second time in five months, a zoo official aid Friday. Low levels of the virus were detected in Achara's blood in a round of testing done this week. Each of the zoo's five elephants is tested weekl
“The risk to the wider population is negligible as the tick that carries CCHF is not established in the UK and it cannot survive here.”
Ed Rybicki's insight:
“The risk to the wider population is negligible as the tick that carries CCHF is not established in the UK and it cannot survive here.” And it is endemic where I live, and I've never met anyone who is even seropositive?
Ebola is 40 today! The discovery of filoviruses: Marburg and Ebola Marburg virus In 1967, the world was introduced to a new virus: thirty-one people in Marburg and Frankfurt in Germany, and Belgrade in the then Yugoslavia, became infected in a linked outbreak with a novel haemorrhagic fever agent. Twenty-five of them were laboratory workers associated…
Viral hepatitis is a leading cause of death and disability worldwide. Unlike most communicable diseases, the absolute burden and relative rank of viral hepatitis increased between 1990 and 2013. The enormous health loss attributable to viral hepatitis, and the availability of effective vaccines and treatments, suggests an important opportunity to improve public health.
Diabetes and its concurrent complications impact a significant proportion of the population of the US and create a large financial burden on the American health care system. FDA-approved maggot debridement therapy (MDT), the application of sterile laboratory-reared Lucilia sericata (green bottle fly) larvae to wounds, is a cost-effective and successful treatment for diabetic foot ulcers and other medical conditions. Human platelet derived growth factor-BB (PDGF-BB) is a secreted dimeric peptide growth factor that binds the PDGF receptor. PDGF-BB stimulates cell proliferation and survival, promotes wound healing, and has been investigated as a possible topical treatment for non-healing wounds. Genetic engineering has allowed for expression and secretion of human growth factors and other proteins in transgenic insects. Here, we present a novel concept in MDT technology that combines the established benefits of MDT with the power of genetic engineering to promote healing. The focus of this study is to create and characterize strains of transgenic L. sericata that express and secrete PDGF-BB at detectable levels in adult hemolymph, whole larval lysate, and maggot excretions/ secretions (ES), with potential for clinical utility in wound healing. We have engineered and confirmed transgene insertion in several strains of L. sericata that express human PDGF-BB. Using a heat-inducible promoter to control the pdgf-b gene, pdgf-b mRNA was detected via semi-quantitative PCR upon heat shock. PDGF-BB protein was also detectable in larval lysates and adult hemolymph but not larval ES. An alternative, tetracycline-repressible pdgf-b system mediated expression of pdgf-b mRNA when maggots were raised on diet that lacked tetracycline. Further, PDGF-BB protein was readily detected in whole larval lysate as well as larval ES. Here we show robust, inducible expression and production of human PDGF-BB protein from two conditional expression systems in transgenic L. sericata larvae. The tetracycline-repressible system appears to be the most promising as PDGF-BB protein was detectable in larval ES following induction. Our system could potentially be used to deliver a variety of growth factors and anti-microbial peptides to the wound environment with the aim of enhancing wound healing, thereby improving patient outcome in a cost-effective manner.
Ed Rybicki's insight:
Now THAT'S cool! Using a VERY old technique, modernised with maggots that make a HGF!
THURSDAY, Sept. 22, 2016 (HealthDay News) -- An experimental DNA-based vaccine protected monkeys from infection with the birth defects-causing Zika virus, and it has proceeded to human safety trials, researchers report.
Health workers in western Niger are racing to contain an outbreak of Rift Valley fever that has killed at least 21 people over the past month, an aid agency said on Wednesday [21 Sep 2016].
The highly contagious disease, which is transmitted to humans by mosquitoes or close contact with contaminated animals, has infected 52 people in Niger's western region of Tahoua since late August, the country's health ministry said.
The Alliance for International Medical Action (ALIMA) and Niger's health authorities have opened an emergency treatment center, in the region's hardest-hit district of Tchintabaraden, to look after the infected and stop the disease from spreading.
"Unfortunately, the 52 severe cases officially registered at present only represent the tip of the iceberg," ALIMA's medical coordinator Oumarou Maidadji said in a statement.
With no specific treatment or effective human vaccine, Rift Valley fever can cause blindness and severe haemorrhaging, leading the victim to vomit blood or even bleed to death.
Herders and farmers [and veterinarians] are deemed at higher risk of infection from the disease, which can devastate livestock.
Niger's health ministry said people in the Tahoua region, especially pastoralists, should avoid handling meat from infected animals, boil raw milk before consumption, and ensure that the corpses of dead animals are buried carefully.
ALIMA is also working with local partners and doctors to provide a mobile clinic which travels the region to inform the public about the disease and how to prevent it from spreading.
Ed Rybicki's insight:
A real, as opposed to the previous imaginary threat.
A yellow fever outbreak in Angola and Congo has been brought under control by a major vaccination campaign, the World Health Organization’s director of infectious hazard management Sylvie Briand has said. “One (piece of) good news is that this outbreak is under control now. We haven’t had any new cases in Angola since June 23, …
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