Deoxyribozymes: new therapeutics to treat central nervous system disorders

Frontiers in Molecular Neuroscience, 2011 · DOI: 10.3389/fnmol.2011.00025 · Published: September 23, 2011

Simple Explanation

Deoxyribozymes, or DNAzymes, are catalytic DNA molecules being explored as therapeutic agents, especially in neurobiology/neuroscience, an area where they've been rarely used. These DNA enzymes can be designed to target specific mRNA sequences, preventing the production of harmful proteins that contribute to conditions like spinal cord injuries. By altering the inhibitory environment of lesion scars after CNS injuries, deoxyribozymes hold promise for promoting axonal growth and functional recovery in the injured spinal cord.

Study Duration

Not specified

Participants

Not specified

Evidence Level

Review

Key Findings

1
Deoxyribozymes can be designed to cleave specific mRNA targets, preventing protein translation and potentially altering disease pathways.
2
A deoxyribozyme targeting xylosyltransferase-1 (XT-1) mRNA has shown promise in promoting sensory axon regeneration in the injured spinal cord by reducing glycosaminoglycan chains in the glial scar.
3
Intravenous administration of a deoxyribozyme targeting c-Jun mRNA (Dz13) showed no significant toxic effects in mice, suggesting potential for systemic delivery.

Research Summary

This mini-review discusses the potential of deoxyribozymes as a therapeutic approach for central nervous system (CNS) disorders, particularly spinal cord injuries (SCIs). Deoxyribozymes are catalytic DNA molecules that can be designed to target and cleave specific mRNA sequences, preventing the production of proteins involved in inhibitory processes after CNS insults. The review highlights the application of deoxyribozymes targeting c-Jun and xylosyltransferase (XT) mRNA, demonstrating their potential to influence neurological disorders and promote axonal regeneration in SCI, respectively.

Practical Implications

Novel Therapeutic Strategy

Deoxyribozymes offer a new approach to treating CNS injuries by altering the inhibitory environment at the lesion site and promoting axonal regeneration.

Targeted Drug Delivery

The ability to design deoxyribozymes to target specific mRNA sequences allows for a more targeted approach to drug development, potentially reducing off-target effects.

Combination Therapies

Deoxyribozymes can be used in combination with other therapeutic approaches to enhance axonal regeneration and improve functional recovery after CNS injuries.

Study Limitations

1
All above mentioned knockdown technologies are incapable to pass the blood–brain or spinal cord-barrier based on their charged DNA or RNA backbone.
2
Immunological status of deoxyribozymes is currently unknown.
3
Explicit information on the half-life of deoxyribozymes in the cell or after systemic administration is also not available.

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