In 2023, the Mass General Brigham Gene and Cell Therapy Institute announced the winners of its first Spark Grant series. This program was created to fund gene and cell therapy projects that demonstrate promising advancements toward clinical applications. The grant awardees included researchers working on cancer, chronic pain, Huntington’s disease, Usher syndrome, and other diseases. Among the recipients was Artur Indzhykulian, MD, PhD, from Mass Eye and Ear, for his project titled “Evaluation of Human Mini-PCDH15s in Human Inner Ear Organoids to Treat USH1F.”
Dr. Indzhykulian's lab uses adeno-associated virus (AAV) vectors, which are small, harmless viruses used by scientists to deliver specific genes into cells. These vectors help study gene functions, correct genetic disorders and develop new treatments by introducing or repairing DNA in targeted cells. Because the PCDH15 gene is too large for typical AAV vectors, they developed mini versions that can restore hearing and balance in mouse models of USH1F. The team plans to design similar human mini-PCDH15s to test whether this approach will work in human inner-ear organoids.
What this means for Usher syndrome: This grant will allow research to progress in Dr. Artur Indzhykulian’s lab to provide proof-of-concept, preclinical evaluation of human mini-PCDH15s.
