Update – Barbara Lee Waszczak has been selected as an honoree!
The human brain – taken for granted every second of our lives. To think, to talk, to walk, eat, breath … we need a fully functioning brain. Day after day we continue to use it, stress it, relax it, grind it. As we continue to age, absently utilizing this wonderfully complex organ, it begins to give up on us – slowly, yet steadily. Sooner to some, later to others, but reduced cranial activity is the reality that all must face. To add to it, neurodegenerative diseases like Parkinson’s and Alzheimer’s plague our lives, the maladies of the 21st century.
Treatment for most central nervous system (CNS) diseases is met with two very broad issues. The first being the absence of curative therapeutics and the presence of medications that only suppress symptoms, and the second being the nearly intractable challenge of the blood-brain barrier. How to permeate the barrier and effectively deliver drugs has puzzled scientists for years
Enter genes. Genes, the central dogma of our being, undergo alterations over time. When genetic mutations take place, abnormalities emerge giving rise to diseases like Alzheimer’s and Parkinson’s. Employing gene therapy to treat CNS related diseases tackles two of the most daunting problems: 1. attacking the problem at the root cause, and 2. eliminating the need for drugs to cross the blood-brain barrier. Gene therapy attempts to restore activity of a gene or silence an overactive gene thereby fixing problems at the core. However, this is not as easy at is sounds.
At Northeastern University, Barbara Lee Waszczak and her team have found a path breaking, revolutionary way of approaching gene therapy in respect to CNS related diseases. The novelty of her invention comes from administering nanoparticles coated with nucleic acids intranasally. Nucleic acids are the building blocks of DNA, and, thus, genes. The administered nanoparticlenucleotide combination forms a sequence holding the code for a protein capable of upregulating the genetic function of the target gene or downregulating it.
The route of administration, i.e., intranasal, and the one nucleotide per nanoparticle concept makes this a pioneering invention with solid contributions to the fields of genetics, therapeutics, personalized medicine, and beyond. Dr. Waszczak has devised a solution to a foundational problem, brought simplicity to undo complexity, and put peace of mind within reach.
Invented Here! Event Information
The 2019 Invented Here! Award Event will be held on November 6th from 6:00 – 9:00pm at the Exchange Conference Center. The evening includes a networking reception, interviews with Featured Inventors, and a chance to connect with forward thinking professionals from across the region.
Come join us as we celebrate Northeastern inventorship and New England innovation! Click here for more information.
Written by Divya Parikh.