via Medical Xpress
A recently developed system for switching on the activity of genes could improve treatments for a broad range of neurological diseases. Esteban Engel, a researcher in viral neuroengineering in the Princeton Neuroscience Institute, and his team have developed new “gene promoters” – which act like switches to turn on gene expression – that promise to broaden the ability to deliver large genes and keep them active for long periods of time.
The research was published online this week in the journal Molecular Therapy: Methods & Clinical Development.
The team is developing these genetic switches for use in gene therapy, the practice of delivering new genes to replace or assist those that are faulty. Gene therapy is a promising strategy for many diseases, including disorders that involve the brain, such as Parkinson’s disease and Alzheimer’s disease.
To carry genes into cells, scientists take advantage of the fact that viruses come equipped with the machinery to gain entry to cells. Over the years, scientists have engineered viruses to deliver genes in ways that are safe and don’t cause disease. One of the viruses commonly used for this is the relatively harmless adeno-associated virus (AAV).
Engel and his team created new gene promoters that turn on genes after they have been transported into neurons – the cells of the brain and nervous system. The team designed their promoters by adopting attributes of promoters found in another class of viruses, the herpes viruses, which persist for years in the body by establishing a chronic infection in the nervous system.
The team’s engineered promoters occupy far less space than existing promoters used in gene therapy, allowing the transport of larger genes or multiple genes. The new promoters are also long-lasting, being less prone to repression or inactivation than most common promoters, so the therapeutic genes are active for long periods of time. These new promoters work with AAV as well as other viral and non-viral gene-delivery systems.
“These new promoters will allow us to deliver larger genes or multiple small genes,” Engel said, “and the genes can remain active for as long as they are needed.”
The Latest Updates from Bing News & Google News
Go deeper with Bing News on:
- Birds with mysterious avian disease found in 14 Northern Virginia localitieson July 6, 2021 at 3:46 pm
The Virginia Department of Wildlife Resources gave an update Tuesday regarding the mortality event affecting birds across multiple states.
- After Timesia Hart Was Diagnosed With a Rare Disease, She Started a Bike Race to Inspire Otherson July 6, 2021 at 12:23 pm
I’m not supposed to be able to walk, let alone ride. I pray that by riding, others will be inspired to keep pushing.” ...
- Law change aims to help contain chronic wasting disease in Minnesotaon July 6, 2021 at 9:35 am
An outbreak of the neurological disease on a farm in Beltrami County earlier this year sparked an outcry because it marked a significant spread of the disease to northern Minnesota and posed an ...
- Alex Agius Saliba elected chairman of European alliance on chronic pain and neurological disorderson July 6, 2021 at 5:20 am
The European Alliance Brain, Mind and Pain brings together 31 associations that work towards the rights of patients suffering from chronic pain and neurological disorders ...
- Alector (ALEC) Up on Tie-Up With Glaxo for Neurological Diseaseson July 4, 2021 at 7:20 pm
Repligen Corporation, GlaxoSmithKline PLC ADR, Trevena Inc, Alector Inc. Read Zacks Investment Research's latest article on Investing.com ...
Go deeper with Google Headlines on:
Go deeper with Bing News on:
- Three-in-one approach boosts the silencing power of CRISPRon July 1, 2021 at 9:00 pm
Cas9-based gene editing system has dramatically advanced the field of bioengineering. However, while fusing transcription activator or repressor to dCas9 protein allows targeted alteration of gene ...
- Insomniac’s Day Trip festival relocates again, days before event, refunds fans for tickets and travelon June 30, 2021 at 11:21 pm
The festival, which was originally scheduled for San Pedro July 3-4 before moving to The Lot at SoFi Stadium in Inglewood, is relocating to San Bernardino.
- First in China! Neurophth announced the first patient has been dosed in G.O.L.D. clinical trial for the gene therapy treatment of LHONon June 28, 2021 at 6:51 am
Novel and one-time intravitreal injection of ND4 gene therapy for the treatment of LHON potentially restored vision; Phase I/II/III clinical trial expected to enroll patients with ...
- First CRISPR-Based Gene Drive Developed in Plantson June 28, 2021 at 5:00 am
Scientists develop the first CRISPR-Cas9-based gene drive in plants which may breed crops better able to withstand drought and disease.
- Cruel World festival — featuring Morrissey, Bauhaus, Blondie, Devo — adds 2nd dateon June 25, 2021 at 11:01 am
The pandemic-canceled Cruel World festival will not only come roaring back in 2022, but promoters today announced a second date featuring the same classic-alternative bill, topped by headliners ...