Specific Delivery Platform Provides Promising Gene Therapy Against Neurodegenerative Diseases

Due to BBB, achieving the delivery of this ASO therapy with systemic dosing and adequate concentrations in the central nervous system (CNS) has been difficult.

‘Specific group of drugs – antisense oligonucleotides (ASOs) are found to efficiently cross the blood-brain barrier with intravenous or subcutaneous dosing when conjugated with cholesterol. This may revolutionize the management of several neurodegenerative diseases.


Innovative ASO Therapy

To overcome the hurdle, the study team has now designed a suitable drug delivery platform of ASO therapy – the DNA/RNA heteroduplex oligonucleotide (HDO) technology that is capable of highly efficient RNA degradation in vivo.

“We found that cholesterol conjugated HDO (Chol-HDO), unlike cholesterol-ASO, efficiently reached the CNS following subcutaneous or intravenous administration in experimental animals. The Chol-HDO platform showed significant dose-dependent target gene reductions with prolonged action in all CNS regions and cell types,” says Tetsuya Nagata, the First author of the study.


The drug efficacy was confirmed across species and against many neurodegenerative diseases such as myotonic dystrophy type 1, Alexander disease, and amyotrophic lateral sclerosis. The drugs ameliorate the diseases at the genetic level by suppressing the production of harmful proteins or non-coding RNAs.

“Our innovative therapeutic platform for blood-to-brain delivery of ASOs may revolutionize the management of neurodegenerative diseases. Future research will help define the specific molecular pathways thus optimizing the delivery of ASO pharmacotherapy to the CNS,” says Takanori Yokota, the senior author of the study.

Source: Medindia


Stay connected with us on social media platform for instant update click here to join our  Twitter, & Facebook

We are now on Telegram. Click here to join our channel (@TechiUpdate) and stay updated with the latest Technology headlines.

For all the latest Health News Click Here 


 For the latest news and updates, follow us on Google News

Read original article here

Denial of responsibility! TechiLive.in is an automatic aggregator around the global media. All the content are available free on Internet. We have just arranged it in one platform for educational purpose only. In each content, the hyperlink to the primary source is specified. All trademarks belong to their rightful owners, all materials to their authors. If you are the owner of the content and do not want us to publish your materials on our website, please contact us by email – [email protected]. The content will be deleted within 24 hours.