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De Novo and Dominantly Inherited SPTAN1 Mutations Cause Spastic Paraplegia and Cerebellar Ataxia.

Liedewei Van de VondelJonathan De WinterDanique BeijerGiulia CoarelliMelanie WayandRobin PalvadeauMartje G PaulyKatrin KleinMaren RautenbergLéna Guillot-NoëlTine DeconinckAtay VuralSibel ErtanOkan DoguHIlmi UysalVesna BrankovicRebecca HerzogAlexis BriceAlexandra DurrStephan KlebeFriedrich StockAlmut Turid BischoffTim W RattayMaría-Jesús SobridoGiovanna De MichelePeter De JongheThomas KlopstockKatja LohmannGinevra ZanniFilippo M SantorelliVincent TimmermanTobias B HaackStephan Züchnernull nullRebecca SchüleGiovanni StevaninMatthis SynofzikAyse Nazli BasakJonathan Baets
Published in: Movement disorders : official journal of the Movement Disorder Society (2022)
We show that SPTAN1 is a relevant candidate gene for ataxia and spastic paraplegia. We suggest that for the mutations identified in this study, disruption of the interlinking of spectrin helices could be a key feature of the pathomechanism. © 2022 International Parkinson and Movement Disorder Society.
Keyphrases
  • cerebral palsy
  • early onset
  • botulinum toxin
  • upper limb
  • copy number
  • genome wide
  • deep learning
  • transcription factor
  • genome wide identification