SMU nanotechnology skilled MinJun Kim and his staff have been awarded a $1.8 million, R01 grant from the Nationwide Institutes of Well being (NIH) for analysis associated to gene remedy – a way that modifies an individual’s genes to deal with or treatment 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 an easier, more practical approach 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 measurement from 1 to 100 nanometers (one billionth of a meter) in measurement. Nanomaterials can happen naturally and can be engineered to carry out particular features, such because the supply of medicine to numerous types of most cancers. Viruses are gentle nanoparticles.
The protein coat surrounding the nucleic acid of a virus 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 approach to ship throughout the human physique a wholesome copy of a gene to exchange or modify a disease-causing one – a course of generally known as viral gene remedy.
Present assessments like ELISA and qPCR cannot inform exactly whether or not viruses are carrying the correct quantity (or any) of the genetic cargo they’re meant to ship, doubtlessly placing sufferers in danger.
“We anticipate that the groundwork laid by this challenge will undeniably rework the way in which nanoscale species, resembling 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 Heart, and Prashanta Dutta at Washington State College are working with lead investigator Kim on the analysis.
Addressing essential issues in viral gene remedy
The staff 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 firms think about AAV to be an important 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 machine and analytical instruments being developed – which Kim known as “next-generation expertise for all-in-one virus characterization” – is called 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 based mostly on how a lot cargo content material is current inside them.
Kim and his staff can be making use of machine-learning, laptop programs that draw inferences and “study” from patterns in knowledge, to the large amount of optical-electrical indicators 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 dear and wasteful,” Kim stated. “Our proposed sensor requires solely minute virus quantities. This challenge will allow my staff to conduct cutting-edge analysis, buying information 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/
