RESEARCH · PROJECT 4
EMERGING
AAV-Based Gene Therapy
for Sensory Disorders
We develop new therapeutics for allergic diseases and chronic pain & itch by combining 16 years of ion channel drug discovery expertise with AAV-based gene therapy.
AAV Gene Therapy
Ion Channels
DRG Neurons
Refractory Sensory Disorders
Cell-Type Specific Delivery
Chronic Pain
Chronic Itch
THE PROBLEM
Different Diseases, One Shared Root
For millions of patients suffering from chronic pain, refractory itch, and severe allergic conditions, current treatments often provide only temporary relief or carry significant side effects. These sensory disorders are frequently treated as separate entities, yet they often share a common origin: the dysregulation of specific ion channels in sensory neurons.
The challenge lies in delivering therapeutic agents directly to these affected cells without disrupting normal physiological functions. Conventional systemic drugs circulate through the entire body, leading to off-target effects and limiting the dosage that can be safely administered to reach the target site in the nervous system.
"Traditional pharmacology often struggles with precision. By targeting the underlying ion channel dysfunction at its cellular source, we move from managing symptoms to addressing the biological driver of sensory disease."
OUR APPROACH
The Right Channel, the Right Cell, the Right Location
We utilize a multi-pronged approach to overcome the limitations of systemic drug delivery, ensuring that therapeutic cargoes are delivered exactly where they are needed to restore sensory balance.
STRATEGY 01
Ion Channel-Targeted Cargo
Developing small molecule or biological modulators that interact specifically with the pore-forming subunits of target ion channels.
STRATEGY 02
Focal AAV Delivery
Utilizing localized viral administration to restrict expression to the specific tissue region affected by the sensory condition.
STRATEGY 03
Cell-Type Specific Promoter Engineering
Engineering genetic payloads with custom promoters that activate therapeutic expression only within particular neuronal subtypes.
RESEARCH VISION
Where This Program Is Headed
Our vision is to build a comprehensive technology platform that bridges the gap between basic neurobiology and clinical application. By integrating high-resolution sensory mapping with innovative delivery systems, we aim to provide long-lasting, cell-specific interventions for patients who currently lack effective treatment options.
PLATFORM DEVELOPMENT
Building the Gene Therapy Platform at SKKU
We are establishing a robust pipeline at SKKU for the rapid screening and validation of AAV vectors optimized for peripheral sensory nerve targeting.
ONGOING COLLABORATION
Joint Research with MGH/Harvard
Collaborative efforts with partners at Massachusetts General Hospital and Harvard Medical School focus on validating our neuro-targeting promoters.
LONG-TERM VISION
From Bench to IND
Our ultimate goal is to translate these laboratory successes into Investigational New Drug (IND) filings, bringing gene therapies to clinical trials.
Interested in this program?
Students and collaborators can learn more about our ongoing research or get in touch directly.