SMU Nanotechnology Skilled Receives $1.8 Million for Analysis Associated to Gene Remedy



SMU nanotechnology skilled MinJun Kim and his workforce have been awarded a $1.8 million, R01 grant from the Nationwide Institutes of Well being (NIH) for analysis associated to gene remedy – a method that modifies an individual’s genes to deal with or remedy illness.

NIH R01 (Analysis Program) grants are extraordinarily aggressive, with fewer than 10 p.c of candidates receiving one. 

The four-year grant will permit Kim, the Robert C. Womack Chair within the Lyle College of Engineering at SMU (Southern Methodist College) and principal investigator of the BAST Lab, to develop a less complicated, more practical option to precisely decide whether or not viruses supposed for gene remedy include their full genetic cargo. 

Nanoparticles are too small to be seen to the bare eye – ranging in measurement from 1 to 100 nanometers (one billionth of a meter) in measurement. Nanomaterials can happen naturally and will also be engineered to carry out particular features, such because the supply of medication to numerous types of most cancers. Viruses are gentle nanoparticles.

The protein coat surrounding the nucleic acid of a virus is known as a capsid. It protects the genetic materials that the virus is carrying. Not with the ability to decide the integrity of the capsid and the quantity of genetic materials it could be defending can result in overdosing or underdosing. That menace is a key barrier to utilizing innocent viruses as a option to ship throughout the human physique a wholesome copy of a gene to switch or modify a disease-causing one – a course of generally known as viral gene remedy.

Current exams like ELISA and qPCR cannot inform exactly whether or not viruses are carrying the correct quantity (or any) of the genetic cargo they’re supposed to ship, probably placing sufferers in danger. 

“We anticipate that the groundwork laid by this venture will undeniably rework the way in which nanoscale species, comparable to viruses and virus-like nanoparticles, are analyzed for cargo content material,” Kim mentioned.

George Alexandrakis on the College of Texas at Arlington, Steven Grey on the College of Texas Southwestern Medical Heart, and Prashanta Dutta at Washington State College are working with lead investigator Kim on the analysis. 

Addressing vital issues in viral gene remedy

The workforce might be testing how precisely a tool they created measures the genetic content material for adeno-associated virus (AAV), a virus encapsulated with single-stranded or double-stranded DNA that has not been discovered to trigger any illnesses in folks. Pharmaceutical corporations contemplate AAV to be a terrific potential vessel for gene remedy. As an example, Luxturna, the primary FDA-approved gene remedy (2017), is an AAV that carries genetic supplies to deal with hereditary blindness.

The system and analytical instruments being developed – which Kim known as “next-generation know-how for all-in-one virus characterization” – is named a bimodal optical-electric plasmonic nanopore sensor.

The sensor will decide the dimensions, efficient cost and deformability of particular person AAVs. Voltage-induced deformability issues, as a result of the form of virus capsids change primarily based on how a lot cargo content material is current inside them. 

Kim and his workforce might be making use of machine-learning, pc techniques that draw inferences and “be taught” from patterns in knowledge, to the huge amount of optical-electrical indicators the nanopore sensor receives, giving higher classifications of whether or not drug-delivering viruses are carrying their supposed disease-fighting genetic supplies.

“Present analytical strategies require utilizing giant quantities of the virus preparation for high quality management, which is dear and wasteful,” Kim mentioned. “Our proposed sensor requires solely minute virus quantities. This venture will allow my workforce to conduct cutting-edge analysis, buying data in state-of-the-art nanotechnology, together with biomechanics and mechanobiology, nanophotonics, nanofabrication, machine studying, bodily virology, and gene supply techniques.”

Supply: https://www.smu.edu/

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