Two preclinical gene therapy advances target neurodegeneration and sensory loss. One team delivered VGLUT3 to inner hair cells and restored hearing in aging mice, highlighting a pathway to address age‑related auditory decline. Separately, researchers used AAV vectors to deliver BDNF and GAS6 to muscle in SOD1G93A ALS mice, slowing disease onset and extending function. The hearing study demonstrates proof‑of‑concept for synaptic restoration in cochlear inner hair cells, while the ALS work shows peripheral delivery of neurotrophic and protective factors can modify disease trajectory in a genetic mouse model. Both studies were reported in peer‑reviewed outlets and presented at scientific meetings. Translation will require vector safety profiling, dose optimization, and biodistribution assessment. These results reinforce AAV’s versatility for CNS and neuromuscular targets but also underscore the need for robust translational pipelines to navigate immune, dosing and manufacturing hurdles.