# Sonogenetic Restoration of Vision for Retinitis Pigmentosa

> **NIH NIH U01** · NANOSCOPE TECHNOLOGIES, LLC · 2024 · $785,380

## Abstract

Retinitis Pigmentosa (RP) often results in legal blindness and is caused by more than 100 genetic mutations,
making its development challenging to address all causes with gene therapy. FDA approval of gene therapy for
LCA addressing RPE65 mutation has sparked great enthusiasm and hope for developing gene therapy.
However, classical gene replacement or gene-editing-based sight-saving therapies are applicable when
photoreceptors do not degenerate. Current systems, however, are limited by poor resolution and retinal
damage over a period. Most current clinical treatments are primarily focused on slowing down the progression
of the disease, as there is neither a cure that can stop the degeneration nor a therapy other than retinal
prostheses that can improve vision loss due to retinal degeneration. However, the electrical stimulation devices
are invasive and require complex implantation surgeries. We have investigated ultrasound stimulation for the
retina as a modality for treating retinal degenerative diseases. However, significant challenges in translating
this technology include (i) the requirement of high-intensity ultrasound and (ii) the lack of cell-specific
stimulation. By sensitizing specific retinal cells (e.g., retinal ganglion cells that are intact in RP) toward
ultrasound using the heterologously-expressed mechanosensitive channel, the sonogenetic stimulation-based
approach may provide an alternative therapeutic modality to restore visual function in the degenerated retina.
Nanoscope has screened and engineered mechanosensitive channels (EMC) from different microbial species,
which upon expression in mammalian cells and retina, allows sonogenetic stimulation by ultrasound with
intensity orders of magnitude lower than that required without EMC sensitization. EMC is activated by any
mechanical force that causes stretch and deformation of the lipid bilayer, such as stretch, pressure/suction,
and ultrasound, as measured by pressure clamp and electrophysiology. RGC-specific EMC delivery combined
with external US stimulation has generated restoration of visual function in the RP mice model. In the proposed
project, we aim to utilize sonogenetic stimulation using ultrasound array transducers for achieving vision
restoration in the photoreceptor-degenerated retina. This goal will be achieved through the following aims: Aim
1: In-vitro assessment of the efficacy of EMC by use of focused ultrasound stimulation, Aim 2: In-vivo
assessment of low-power ultrasound stimulation of EMC-sensitized retina, Aim 3: Sonogenetic restoration of
vision in a rabbit model of Retinitis Pigmentosa. The success of this proposed project will lead to the realization
of a non-invasive sonogenetic combination therapy for vision restoration in retinal degenerative diseases.

## Key facts

- **NIH application ID:** 10850454
- **Project number:** 1U01EY035026-01A1
- **Recipient organization:** NANOSCOPE TECHNOLOGIES, LLC
- **Principal Investigator:** Samarendra Kumar Mohanty
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $785,380
- **Award type:** 1
- **Project period:** 2024-05-01 → 2028-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10850454

## Citation

> US National Institutes of Health, RePORTER application 10850454, Sonogenetic Restoration of Vision for Retinitis Pigmentosa (1U01EY035026-01A1). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10850454. Licensed CC0.

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