Here Come More Gene Therapies -- And More Pricing Debates
Gene therapies like Spark Therapeutics'upcoming Luxturna and Novartis'FDA-approved Kymriah are raising concerns about the rising cost of health care, leading some experts to suggest new ways to price treatments based on the benefits they offer.
CONCLUSION: LPC conditioning prior to LV vector delivery, substantially enhanced the level of conducting airway gene expression after a single gene vector delivery. These results extend the previously established effectiveness of this protocol for producing gene expression in the nasal airways to the lung airways, the primary site of deleterious pathophysiology in CF individuals. PMID: 29236544 [PubMed - as supplied by publisher]
22 –25 January 2018, Miami, United States
Development of an adenovirus vector vaccine platform for targeting dendritic cells, Published online: 15 December 2017; doi:10.1038/s41417-017-0002-1Development of an adenovirus vector vaccine platform for targeting dendritic cells
Alzheimer's disease (AD) is a progressive neurodegenerative disease characterized by synaptic dysfunction and accumulation of abnormal aggregates formed by amyloid‐β peptides or phosphorylated tau proteins. Accumulating evidence suggests that alterations in the buffering capacity of the proteostasis network are a salient feature of AD. The endoplasmic reticulum (ER) is the main compartment involved in protein folding and secretion and is drastically affected in AD neurons. ER stress triggers the activation of the unfolded protein response (UPR), a signal transduction pathway that enforces adaptive programs to recove...
A gene-therapy trial by London researchers has seen remarkable success in finding a long-term cure for hemophilia A.
Jake Omer, 29 from Billericay, Essex, who took part in the Bars Health NHS Trust trial, revealed the gene therapy changed his life and that he now has 'hope for the future'.
A CURE for haemophilia is one step closer after “mind-blowing results” from a gene therapy trial.
A 'cure' for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London. Clinical researchers at Barts Health NHS Trust and Queen Mary University of London have found that over one year on from a single treatment with a gene therapy drug, participants with haemophilia A (the most common type) are showing normal levels of the previously missing protein, and effectively curing them.
How pioneering gene therapy has given a haemophilia patient a new lease of life.
Doctors use gene therapy to correct the defect that causes haemophilia A.