Effects of a ciliary neurotrophic factor (CNTF) small-molecule peptide mimetic in an in vitro and in vivo model of CDKL5 deficiency disorder.
Mottolese. Nicola N; Loi. Manuela M; Trazzi. Stefania S; Tassinari. Marianna M;...
The peptide P021 helped brain cells grow and survive in a dish made from CDKL5‑deficient cells, but when given to CDKL5‑deficient mice it did not raise BDNF levels, fix brain structure, or meaningfully improve behavior. More work is needed to see if starting treatment earlier or giving it longer could work.