Unmet Need
Globally, millions of patients are affected by diseases caused when mutations affect the normal function of voltage gated sodium channels (Nav). These mutations have been implicated in several human genetic diseases such as hypokalemic periodic paralysis, myotonia and Brugada syndrome. Myotopic Dystrophy alone affects at least 1 in 8,000 people worldwide. Therefore, there is high demand for research, diagnostic, and therapeutic tools that target voltage gated sodium channels. Despite the physiological importance of these channels, development of antibodies specific for the different Nav isoforms has been challenging.
Technology Overview
Johns Hopkins researchers have produced, purified and characterized two nanobodies, Nb17 and Nb82, which recognize human voltage gated Sodium Channels (Nav 1.4 and Nav 1.5). Nav 1.4 and Nav1.5 are the principal Nav isoforms in skeletal and cardiac muscle, respectively. These nanobodies recognize Nav 1.4 and Nav 1.5 with nM or higher affinity and without cross-reactivity tested to other isoforms.
Applications for these nanobodies include, molecular Nav visualization agents (WB, ELISA, live cell imaging), bait proteins to capture and purify Navs from cell lysates, Nav channel modulators for tissue-specific targeting in health and disease and crystallization chaperones.
Stage of Development
The technology is ready to be commercialize as a molecular probe for research or diagnostics.
Publication
N/A