Targeted treatment for autosomal dominant leukodystrophy: promising in vitro results

Lesen Sie diesen Artikel auf Deutsch.

Takeaway

  • Allele-specific silencing by RNA interference (ASP-siRNA) ameliorated protein overexpression and disease-specific phenotypic abnormalities in an in vitro model of autosomal dominant adult-onset demyelinating leukodystrophy (ADLD) due to lamin B overexpression (LMNB1).

Why this matters

  • ASP-siRNA shows promise as a therapeutic option for this fatal condition and could inform development of similar targeted treatments for other pathological conditions linked to gene copy number variations.

Mochten Sie mehr lesen?

Möchten Sie mehr lesen?

Loggen Sie sich ein oder registrieren Sie sich, um Zugang zu allen Inhalten von Neurodiem zu erhalten.

Sie haben bereits einen Account? Einloggen

International Medical Press is a global provider of independent medical education. Its mission is to provide healthcare professionals with high-quality, trusted medical information with the aim of helping optimize patient care.

No responsibility is assumed by International Medical Press for any injury and/or damage to persons or property through negligence or otherwise, or from any use or operation of any methods, products, instructions, or ideas contained in the material herein. Because of rapid advances in the medical sciences, International Medical Press recommends that independent verification of diagnoses and drug dosages should be made. The opinions expressed do not reflect those of International Medical Press or the sponsor. International Medical Press assumes no liability for any material contained herein.