A morpholino antisense oligonucleotide rescues type I and type III SMA mice

Keyphrases

• Antisense 12%
• Antisense Oligonucleotides 25%
• Clinical Trials 25%
• Day 14 12%
• Dystrophy 12%
• Exon 7 Inclusion 12%
• Homozygous Deletion 12%
• In Vitro Data 12%
• In Vivo Validation 12%
• Morpholino 12%
• Morpholino Antisense Oligonucleotide 100%
• Motor Neuron Degeneration 12%
• Muscle Weakness 12%
• Neonatal Mice 12%
• Oligonucleotides 12%
• Post-neonatal 12%
• Severe Disease 12%
• Silencer 12%
• Single nucleotide 12%
• SMN1 Gene 25%
• SMN2 25%
• Spinal muscular Atrophy 100%
• Survival Motor Neuron 25%
• Systemic Delivery 12%
• Tail Necrosis 12%
• Therapeutic Efficacy 12%
• Therapeutic Strategies 12%
• Therapeutic Window 25%
• Truncated Transcripts 12%

Medicine and Dentistry

• Antisense 16%
• Antisense Oligonucleotide 100%
• Clinical Study 16%
• Clinical Trial 33%
• Combination Therapy 16%
• Dystrophy 16%
• Exon 16%
• In Vitro 16%
• Intron 16%
• Kugelberg Welander Disease 100%
• Muscle Weakness 16%
• Necrosis 16%
• Nerve Cell Degeneration 16%
• Oligonucleotide 16%
• Spinal Muscular Atrophy 100%
• Survival Motor Neuron Protein 16%
• Tail 16%
• Therapeutic Window 33%
• Werdnig Hoffmann Disease 16%

Biochemistry, Genetics and Molecular Biology

• Antisense 100%
• Clinical Study 16%
• Clinical Trial 33%
• Exon 16%
• Intron 16%
• Morpholino 100%
• Motor Neuron 33%
• Nerve Cell Degeneration 16%
• Oligonucleotide 100%
• SMN2 33%
• Survival of Motor Neuron 16%