Current understanding of and emerging treatment options for spinal muscular atrophy with respiratory distress type 1 (SMARD1).

Perego, Martina G L; Galli, Noemi; Nizzardo, Monica; Govoni, Alessandra; Taiana, Michela; Bresolin, Nereo; Comi, Giacomo P; Corti, Stefania

Perego, Martina G L; Galli, Noemi; Nizzardo, Monica; Govoni, Alessandra; Taiana, Michela; Bresolin, Nereo; Comi, Giacomo P; Corti, Stefania.

Affiliation

Perego MGL; Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Via Francesco Sforza 35, 20122, Milan, Italy.
Galli N; Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Via Francesco Sforza 35, 20122, Milan, Italy.
Nizzardo M; Neurology Unit, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Francesco Sforza 35, 20122, Milan, Italy.
Govoni A; Neurology Unit, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Francesco Sforza 35, 20122, Milan, Italy.
Taiana M; Neurology Unit, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Francesco Sforza 35, 20122, Milan, Italy.
Bresolin N; Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Via Francesco Sforza 35, 20122, Milan, Italy.
Comi GP; Neurology Unit, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Francesco Sforza 35, 20122, Milan, Italy.
Corti S; Neuroscience Section, Department of Pathophysiology and Transplantation (DEPT), Dino Ferrari Centre, University of Milan, Via Francesco Sforza 35, 20122, Milan, Italy.

Cell Mol Life Sci ; 77(17): 3351-3367, 2020 Sep.

Article in En | MEDLINE | ID: mdl-32123965

Subject(s)

DNA-Binding Proteins/genetics; Muscular Atrophy, Spinal/pathology; Respiratory Distress Syndrome, Newborn/pathology; Transcription Factors/genetics; Animals; Cell- and Tissue-Based Therapy; DNA-Binding Proteins/chemistry; DNA-Binding Proteins/metabolism; Genetic Therapy; Humans; Muscular Atrophy, Spinal/complications; Muscular Atrophy, Spinal/genetics; Muscular Atrophy, Spinal/therapy; Neural Stem Cells/cytology; Neural Stem Cells/metabolism; Neural Stem Cells/transplantation; Respiratory Distress Syndrome, Newborn/complications; Respiratory Distress Syndrome, Newborn/genetics; Respiratory Distress Syndrome, Newborn/therapy; Ribosomes/chemistry; Ribosomes/metabolism; Survival of Motor Neuron 1 Protein/genetics; Transcription Factors/chemistry; Transcription Factors/metabolism

Key words

Distal hereditary motor neuropathy type 6; Gene therapy; IGHMBP2; Motor neuron disease; Oligonucleotides; SMARD1

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Full text: 1 Database: MEDLINE Main subject: Respiratory Distress Syndrome, Newborn / Transcription Factors / Muscular Atrophy, Spinal / DNA-Binding Proteins Type of study: Prognostic_studies Limits: Animals / Humans Language: En Year: 2020 Type: Article

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