SMU nanotechnology skilled MinJun Kim and his crew 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 % of candidates receiving one.
The four-year grant will enable Kim, the Robert C. Womack Chair within the Lyle Faculty of Engineering at SMU (Southern Methodist College) and principal investigator of the BAST Lab, to develop a less complicated, simpler method to precisely decide whether or not viruses meant for gene remedy include their full genetic cargo.
Nanoparticles are too small to be seen to the bare eye – ranging in dimension from 1 to 100 nanometers (one billionth of a meter) in dimension. Nanomaterials can happen naturally and may also be engineered to carry out particular capabilities, such because the supply of medicine to varied types of most cancers. Viruses are gentle nanoparticles.
The protein coat surrounding the nucleic acid of a virus is named 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 might be defending can result in overdosing or underdosing. That menace is a key barrier to utilizing innocent viruses as a method to ship throughout the human physique a wholesome copy of a gene to interchange or modify a disease-causing one – a course of referred to as viral gene remedy.
Current assessments like ELISA and qPCR cannot inform exactly whether or not viruses are carrying the correct amount (or any) of the genetic cargo they’re meant to ship, probably placing sufferers in danger.
“We anticipate that the groundwork laid by this venture will undeniably rework the best way nanoscale species, comparable to viruses and virus-like nanoparticles, are analyzed for cargo content material,” Kim stated.
George Alexandrakis on the College of Texas at Arlington, Steven Grey on the College of Texas Southwestern Medical Middle, and Prashanta Dutta at Washington State College are working with lead investigator Kim on the analysis.
Addressing essential issues in viral gene remedy
The crew can 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 individuals. Pharmaceutical corporations think about AAV to be an ideal potential vessel for gene remedy. As an illustration, Luxturna, the primary FDA-approved gene remedy (2017), is an AAV that carries genetic supplies to deal with hereditary blindness.
The gadget and analytical instruments being developed – which Kim referred to as “next-generation know-how for all-in-one virus characterization” – is called a bimodal optical-electric plasmonic nanopore sensor.
The sensor will decide the scale, efficient cost and deformability of particular person AAVs. Voltage-induced deformability issues, as a result of the form of virus capsids change based mostly on how a lot cargo content material is current inside them.
Kim and his crew can be making use of machine-learning, laptop programs that draw inferences and “be taught” from patterns in information, to the huge amount of optical-electrical alerts the nanopore sensor receives, giving higher classifications of whether or not drug-delivering viruses are carrying their meant disease-fighting genetic supplies.
“Present analytical strategies require utilizing massive quantities of the virus preparation for high quality management, which is expensive and wasteful,” Kim stated. “Our proposed sensor requires solely minute virus quantities. This venture will allow my crew 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 programs.”
Supply: https://www.smu.edu/
