Results 111 to 120 of about 9,361 (152)

Ptbp2 re-expression rescues axon growth defects in Smn-deficient motoneurons. [PDF]

Front Mol Neurosci
Salehi S, Zare A, Gandhi G, Sendtner M, Briese M.   +4 more
europepmc   +1 more source

Taldefgrobep Alfa and the Phase 3 RESILIENT Trial in Spinal Muscular Atrophy. [PDF]

Int J Mol Sci
Servais L, Lair LL, Connolly AM, Byrne BJ, Chen KS, Coric V, Qureshi I, Durham S, Campbell DJ, Maclaine G, Marin J, Bechtold C.   +11 more
europepmc   +1 more source

Hypothetical Pathogenetic Model of Membranous Nephropathy. [PDF]

Int J Mol Sci
Zdravkova I, Tilkiyan E, Bozhkova D, Kuskunov T, Ronchev Y, Kirilov B.   +5 more
europepmc   +1 more source

The triple code model for advancing research in rare and undiagnosed diseases beyond the base pairs. [PDF]

Epigenomics
Lomberk G, Urrutia R.
europepmc   +1 more source

Physiological Features of the Neural Stem Cells Obtained from an Animal Model of Spinal Muscular Atrophy and Their Response to Antioxidant Curcumin. [PDF]

Int J Mol Sci
Adami R, Pezzotta M, Cadile F, Cuniolo B, Rovati G, Canepari M, Bottai D.   +6 more
europepmc   +1 more source

BRD9 functions as a methylarginine reader to regulate AKT-EZH2 signaling. [PDF]

Sci Adv
Yin S, Brobbey C, Ball LE, Fu T, Sprague DJ, Gan W.   +5 more
europepmc   +1 more source

Performance of cellulose-based card for direct genetic testing of spinal muscular atrophy. [PDF]

BMC Biotechnol
Wijaya YOS, Ar Rochmah M, Nurputra DK, Farmawati A.   +3 more
europepmc   +1 more source

Keep calm and reboot - how cells restart transcription after DNA damage and DNA repair. [PDF]

FEBS Lett
Donnio LM, Giglia-Mari G.
europepmc   +1 more source

Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophy. [PDF]

J Clin Invest
Leow DM, Ng YK, Wang LC, Koh HW, Zhao T, Khong ZJ, Tabaglio T, Narayanan G, Giadone RM, Sobota RM, Ng SY, Teo AKK, Parson SH, Rubin LL, Ong WY, Darras BT, Yeo CJ.   +16 more
europepmc   +1 more source

Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts. [PDF]

Mol Ther Methods Clin Dev
Signoria I, Zwartkruis MM, Geerlofs L, Perenthaler E, Faller KME, James R, McHale-Owen H, Green JW, Kortooms J, Snellen SH, Asselman FL, Gillingwater TH, Viero G, Wadman RI, van der Pol WL, Groen EJN.   +15 more
europepmc   +1 more source