RNA therapies explained

RNA-Targeting Therapies: Translating Hope

On July 20, 2016, the UK health regulator NICE published final guidexcitement for read-through therapies in some camps. An alternative apance recommending the drug ataluren for children with Duchenne muscular dystrophy (DMD). This was a momentous announcement for those who had campaigned vociferously for its approval in the UK. DMD is a rare X-linked disorder that causes progressive muscular we...

Explained Bioinformatics explained : RNA secondary structure partition function , calculation and use

RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a fatal motor disease in adults. Its pathophysiology remains mysterious, but tremendous advances have been made with the discovery of the most frequent mutations of its more common familial form linked to the C9ORF72 gene. Although most cases are still considered sporadic, these genetic mutations have revealed the role of RNA production, processing and tra...

RNA interference therapies – a RISCy business?

This paradigm has suggested a number of therapeutic approaches. For example, gene therapies seek to treat genetic disorders through augmenting the patient’s own complement of genes by the introduction of an exogenous gene, and antisense therapies seek to treat disease by specifically obstructing the expression of a disease-associated gene. However, neither of these approaches has been particula...

RNA Interference Therapies for an HIV-1 Functional Cure

HIV-1 drug therapies can prevent disease progression but cannot eliminate HIV-1 viruses from an infected individual. While there is hope that elimination of HIV-1 can be achieved, several approaches to reach a functional cure (control of HIV-1 replication in the absence of drug therapy) are also under investigation. One of these approaches is the transplant of HIV-1 resistant cells expressing a...