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Gene Therapy for Dementia - Research & Industry Pipelines.

Given that this week is the Alzhemier's Society UK Action Week, it got me thinking about the actions we - as in industry and research - are taking towards finding cures for the causes of dementia.

Lately, with the work we here at Cellix have been doing, I've been looking a lot into the fascinating field of the potential applications of gene editing. With the juggernaut that is CRISPR, it seems that there is no limit to what gene editing can do - that is, in theory.

The reality, as always, is somewhat more complicated. Despite years of investment, history has shown us that diseases causing dementia have typically been difficult to develop drugs for.

So my question became; where are we in terms of gene therapy for these diseases now?


Dementia is a group of symptoms which have touched the lives of many, and has several disease causes. Symptoms can include psychological and cognitive changes, including memory loss, difficulty in communication and cognitive functioning, confusion, personality changes and hallucinations (source: Mayo Clinic).

Causes of dementia include Alzheimer's disease, Huntington's, Parkinson's, vascular dementia, Lewy body dementia and frontotemporal dementia among others.

Currently, there are no therapies which the reverse these diseases entirely, though drugs to halt their progression have improved hugely.

Not all diseases causing dementia are suitable to target using gene therapy - for example, vascular dementia (a result of damage brain blood vessels). While some diseases are associated with a higher incidence of vascular dementia, it's disparate causes make it difficult to target with gene therapy.

However, some other causes of dementia are viable targets for gene therapy. Many diseases causing dementia have the common theme of protein aggregation as a result of misfolding - these include Alzheimer's, Parkinson's, Huntington's, and some prion diseases such as Creutzfeldt-Jakob disease.

Gene therapies for these diseases typically aim to prevent this protein misfolding or aggregation, and are often delivered to the brain using modified viruses.


Alzheimer's is the most common cause of dementia. It is a type of tauopathy - an accumulation of tau proteins. Currently there are 5 FDA-approved drugs for Alzheimer's patients - but these tackle symptoms where we want a cure.

Alzheimer's Disorder

Image: abnormal proteins in the brain cause neurological symptoms. Source: BrightFocus Foundation.

Industry has had a few knocks in this field; so far, 99.6% of Alzheimer's drugs that have made it to late-stage clinical trials have so far failed. Gene therapy may offer the inroads the pharma companies desire.