NINDS launches network to make treatments for extremely-uncommon neurological ailments

NINDS launches network to make treatments for extremely-uncommon neurological ailments
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To the Editor — Uncommon and extremely-uncommon ailments have an impact on hundreds and hundreds of oldsters worldwide. When treatments exist, they’re most incessantly directed on the symptomatic degree in preference to on the mechanistic basis of the disease. Then all another time, due to many uncommon disorders stem from single-gene adaptations, they is at possibility of be addressable the usage of with out be conscious maturing genetic technologies, a lot like antisense oligonucleotides, exiguous interfering RNAs, gene treatment and gene editing; indeed, a Commentary to your June discipline highlighted a now not-for-income initiative to tackle extremely-uncommon ailments the usage of disease-editing antisense oligonucleotides1. Though gene-basically based totally treatment for neurogenetic disorders stays in its infancy (with present questions linked to safety and efficacy), it can perchance perchance also merely offer therapeutic alternatives for instances that on the 2d have few or no present treatment alternatives. In contrast background, the US Nationwide Institute of Neurological Disorders and Stroke (NINDS) is launching a recent program — the Ultra-uncommon Gene-basically based totally Therapy (URGenT) network — that can give a enhance to the pattern of gene-basically based totally treatments for uncommon and extremely-uncommon ailments. The expectation is that such tailored treatments will result in improved affected person outcomes for these ailments.

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Fig. 1: Overview of the URGenT network.


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  1. The Nationwide Institute of Neurological Disorders and Stroke, Nationwide Institutes of Properly being, Bethesda, MD, USA

    Nina F. Schor, Amir P. Tamiz, Walter J. Koroshetz, Julia L. Bachman, Chris Boshoff, Janice Cordell, Neel T. Dhruv, Amelie Gubitz, Adam L. Hartman, Genevieve E. Lind, Codrin I. Lungu, Jill A. Morris, Glen H. Nuckolls, Shantadurga Rajaram, Ranga Rangarajan, Rebecca A. Roof, Mario H. Skiadopoulos, Natalia Strunnikova, Ling M. Wong, Clinton B. Wright & Ann-Marie Broome


NINDS Ultra-Uncommon Gene-basically based totally Therapy (URGenT) Working Community

  • Julia L. Bachman
  • , Chris Boshoff
  • , Ann-Marie Broome
  • , Janice Cordell
  • , Neel T. Dhruv
  • , Amelie Gubitz
  • , Adam L. Hartman
  • , Walter J. Koroshetz
  • , Genevieve E. Lind
  • , Codrin I. Lungu
  • , Jill A. Morris
  • , Glen H. Nuckolls
  • , Shantadurga Rajaram
  • , Ranga Rangarajan
  • , Rebecca A. Roof
  • , Nina F. Schor
  • , Mario H. Skiadopoulos
  • , Natalia Strunnikova
  • , Amir P. Tamiz
  • , Ling M. Wong
  •  & Clinton B. Wright

Corresponding author

Correspondence to
Ann-Marie Broome.

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Competing interests

The authors record no competing interests.

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Schor, N.F., Tamiz, A.P., Koroshetz, W.J. et al. NINDS launches network to make treatments for extremely-uncommon neurological ailments.
Nat Biotechnol (2021).

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