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New approach for oligonucleotide-mediated exon skipping in neurodegenerative diseases

Scientists at the Leiden University Medical Center have developed a new approach for antisense oligonucleotide-mediated exon skipping in neurodegenerative diseases. This method allows for the removal of exons containing either the proteolytic cleavage site or the mutation in the coding region of the gene that is causative for the neurodegeneration.

There are several neurodegenerative diseases that are caused by cleavage of a protein by proteolytic cleavage, resulting in a toxic protein fragment that causes neurodegeneration. Other neurodegenerative diseases are caused by a mutation in the coding region of a gene, resulting in a mutated protein that has gained a toxic function as a result of this mutation. Currently there is no cure on the market for these diseases. Current treatments are symptomatic.

Partner companies are now sought for research collaborations in this field, and licensing of key technologies available at the institutions. Specifically we are looking for companies with a franchise in the treatment of neurodegenerative disorders.

Key benefits

  • Potential cure for certain neuro-degenerative diseases
  • Truncated form of the protein retained
  • Prevents neurodegeneration by reducing the pathogenic form of the target protein instead of preventing down-stream toxic effects
  • The AONs cannot cross the blood brain barrier, thus reducing the chances of unwanted side effects by not entering the periphery


Treatment for Spinocerebellar ataxia 3 (SCA3) and Dentatorubral-pallidoluysian atrophy (DRPLA)

Development stage

Further details available upon request

Luris reference number


Patent status

National phase

Data available on request

Non-confidential and confidential presentations

Further information

Frits Fallaux Knowledge Broker (LU) +31-71-527 2517