Axon Regeneration After Brain or Spinal Cord Injury

The invention is directed to modification of a particular sugar by the enzyme arylsulfatase B (ARSB), which results in axon regeneration.

Following traumatic brain or spinal cord injury, glial scars prevent regeneration of axons. Chondroitin sulfate proteoglycans (CSPGs) are major components of glial scars. CSPGs are made of a protein core containing glycosaminoglycan (GAG) sugar side chains, which, when sulfated, are responsible for the inhibitory activity of glial scars. Specifically, NIH researchers have shown that the 4-sulfate unit on a certain sugar on GAG is responsible for inhibiting axon regrowth and, when the 4-sulfate unit is reduced, axon regrowth is observed. Moreover, removal of this 4-sulfate unit by the ARSB enzyme promotes axon regrowth.

As a potential therapy for spinal cord injuries, researchers developed a vector expressing ARSB and demonstrated that this vector promotes axon regeneration when injected into the spinal cord of a mouse.

Potential Commercial Applications: Competitive Advantages:
  • Treatment of brain and spinal cord injury
  • Treatment of other CNS injuries, including stroke
  • Treatment of heart attack
  • There are no existing products for treatment of traumatic spinal cord injury
  • ARSB is already approved for treatment of Mucopolysaccharoidosis VI, a lysosomal storage disease

Development Stage:
  • Early-stage
  • In vitro data available
  • In vivo data available (animal)


Herbert Geller (NHLBI)  ➽ more inventions...

Yasuhiro Katagiri (NHLBI)  ➽ more inventions...

Intellectual Property:
US Pat: - issued -
US Application No. 61/705,555 filed on 2012-09-25
PCT Application No. PCT/US13/061693 filed on 2013-09-25

Wang H, et al. PMID 18768934

Collaboration Opportunity:

The NHLBI is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize the use of ARSB in axonal regeneration after brain or spinal cord injury using animal models. For collaboration opportunities, please contact Denise Crooks, Ph.D. at 301-435-0103 or

Licensing Contact:
Denise Crooks,
Phone: 301-435-0103

OTT Reference No: E-214-2012-0
Updated: Nov 21, 2012