Possible Optic Nerve Rejuvenation
ER-100 is an investigational gene therapy to reprogram optic nerve cells that was developed by Life Biosciences. It is currently undergoing phase I safety clinical trials in humans.
How it works:
Positive results in mice:
A 2020 study, led by Dr. David Sinclair's team and Yuancheng Lu at Harvard, proved that treating adult mice with an AAV vector expressing Oct4, Sox2, and Klf4 (OSK) could promote optic nerve regeneration after injury, reverse glaucoma-induced damage, and restore visual function in aged animals.
Positive results in nonhuman primates:
Laser-induced ischemic optic neuropathy nerve damage in primates saw a significant reduction in vision deficits, alongside a marked increase in surviving healthy optic nerve axon bundles after receiving the inducible OSK treatment.
How it works:
- The Viral Delivery System: The therapy utilizes a modified, harmless adeno-associated virus (AAV) vector. It is delivered via a single intravitreal injection directly into the eye.
- The Payload (OSK Genes): The virus carries genetic instructions for three specific transcription factors: OCT4, SOX2, and KLF4 (collectively called OSK). These are three of the four Nobel Prize-winning "Yamanaka factors" used to reset mature cells back into stem cells.
- The Doxycycline "On/Off" Switch: Reprogramming adult neurons completely into stem cells is highly dangerous because it can cause tumors. To prevent this, ER-100 features a controlled, switchable system. The OSK genes only activate when the patient takes the common antibiotic doxycycline.
Positive results in mice:
A 2020 study, led by Dr. David Sinclair's team and Yuancheng Lu at Harvard, proved that treating adult mice with an AAV vector expressing Oct4, Sox2, and Klf4 (OSK) could promote optic nerve regeneration after injury, reverse glaucoma-induced damage, and restore visual function in aged animals.
Positive results in nonhuman primates:
Laser-induced ischemic optic neuropathy nerve damage in primates saw a significant reduction in vision deficits, alongside a marked increase in surviving healthy optic nerve axon bundles after receiving the inducible OSK treatment.