[Treating hereditary neuropathies : a dream come true?]

Area of Science:

• Genetics and Neurology
• Molecular Therapeutics

Background:

• Hereditary neuropathies are increasingly benefiting from novel therapeutic strategies.
• Established treatments for hereditary transthyretin amyloidosis (hTTR) and porphyria utilize antisense oligonucleotides (ASO) and small interfering RNA (siRNA).

Purpose of the Study:

• To review recent therapeutic advances in hereditary neuropathies.
• To explore the potential of mRNA and DNA-targeting treatments for conditions like Charcot-Marie-Tooth disease (CMT).

Main Methods:

• Review of current antisense oligonucleotide (ASO) and small interfering RNA (siRNA) therapies.
• Evaluation of emerging CRISPR-Cas9 genomic editing approaches targeting the TTR gene for hTTR.
• Assessment of ongoing research into novel mRNA and DNA-targeting treatments for other hereditary neuropathies.

Main Results:

• Commercial availability of ASO and siRNA treatments for hTTR and porphyria.
• Successful development and patient testing of CRISPR-Cas9 gene editing for hTTR.
• Active evaluation of innovative therapies for additional hereditary neuropathies, including CMT.

Conclusions:

• Significant therapeutic progress has been made in treating hereditary neuropathies.
• mRNA and DNA-based therapies, including gene editing, represent a promising frontier for managing these debilitating neurological disorders.