Lecture
Alternative Strategies for SMA Gene Therapy
Description
This lecture discusses alternative strategies for spinal muscular atrophy (SMA) gene therapy, including the use of antisense oligonucleotides to inhibit exon 8 splicing, enhancing exon 8 inclusion to improve exon 7 levels, and exploring genome editing through homology-directed repair of the SMN2 gene. The potential of non-homologous repair and the limitations of the CRISPR-Cas9 system in SMA gene therapy are also addressed.
In MOOCs (6)
Neuroscience Reconstructed: Cell Biology
This course will provide the fundamental knowledge in neuroscience required to
understand how the brain is organised and how function at multiple scales is
integrated to give rise to cognition and beh
Neuroscience Reconstructed: Cell Biology
This course will provide the fundamental knowledge in neuroscience required to
understand how the brain is organised and how function at multiple scales is
integrated to give rise to cognition and beh
Neuroscience Reconstructed: Genetics and Brain Development
This course will provide the fundamental knowledge in neuroscience required to
understand how the brain is organised and how function at multiple scales is
integrated to give rise to cognition and beh
Neuroscience Reconstructed: Genetics and Brain Development
This course will provide the fundamental knowledge in neuroscience required to
understand how the brain is organised and how function at multiple scales is
integrated to give rise to cognition and beh
Neuroscience Reconstructed: Proteomics
This course will provide the fundamental knowledge in neuroscience required to
understand how the brain is organised and how function at multiple scales is
integrated to give rise to cognition and beh
Instructor
esse mollit
Sit nisi incididunt velit reprehenderit. Eu consequat nisi minim laboris fugiat laborum magna culpa ea ea pariatur commodo. Labore exercitation nostrud culpa in excepteur ex incididunt excepteur. Anim minim sunt nulla minim duis. Id in reprehenderit magna et duis labore commodo nisi mollit sint in ad. Ex aliqua aute est commodo. Laborum sint irure duis incididunt laborum.
Official source
About this result
This page is automatically generated and may contain information that is not correct, complete, up-to-date, or relevant to your search query. The same applies to every other page on this website. Please make sure to verify the information with EPFL's official sources.
Ontological neighbourhood
Related lectures (38)
CRISPR-Based Tools & Diagnostics
Explores CRISPR-based tools and diagnostics for faster, simpler, and cheaper testing methods with high specificity and sensitivity.
CRISPR-based Diagnostics
Covers the history and mechanisms of CRISPR-based tools and diagnostics, including advanced concepts like the SHERLOCK detection assay.
Genome Editing: CRISPR-Cas Applications
Explores the applications of CRISPR-Cas in genome editing, focusing on engineering bacterial genomes, curing genetic diseases, guide RNA simplicity, Cas9 specificity, DNA damage mechanisms, and base editing.
Types of Viral VectorsMOOC: Neuroscience Reconstructed: Cell Biology
Explores the properties of commonly used viral vectors for gene therapy.
Cellular Immunity: Danger Signals and Response Activation
Explores the cellular innate immunity and the activation of the immune response through analogies of naked people in the street representing different cellular threats.