Notes in LE015 Therapeutic Opportunities in Modern Genetics

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Status	Last Update	Fields
Published	10/14/2024	What is gene augmentation?
Published	10/14/2024	What is gene silencing?
Published	10/14/2024	What are transposons?
Published	10/14/2024	Transposon movement can generate {{c1::disease alleles}}
Published	10/14/2024	The {{c1::piRNA pathway}} is used to silence transposons in germline
Published	10/14/2024	Identify the differences between the RNAi pathway and the miRNA pathway
Published	10/14/2024	What is gene editing?
Published	10/14/2024	{{c1::CRISPR-Cas9}} is a common strategy used to introduce targeted double strand breaks
Published	10/14/2024	How is CRISPR used to treat Duchenne Muscular Dystrophy (DMD)?
Published	10/14/2024	Describe CRISPR base editing.
Published	10/14/2024	Describe CRISPR prime editing.
Published	10/14/2024	What is dCas9?
Published	10/14/2024	What are the two modes for delivering CRISPR to diseased tissues?
Published	10/14/2024	What is gene therapy?
Published	10/14/2024	What are the three central challenges to enginering genetic therapeutics?
Published	10/14/2024	Angelman syndrome, a neurodevelopmental disorder, is caused by silencing of {{c1::maternal}} UBE3A, the only allele expressed in the brain
Published	10/14/2024	How is the paternal copy of UBE3A silenced in the brain?
Published	10/14/2024	Describe how the antisense oligo therapy for Angelman syndrome re-expresses the paternal UBE3A copy.
Published	10/14/2024	Describe the gene augmentation strategies used to treat spinal muscular atrophy.
Published	10/14/2024	RNA interference can lead to {{c1::gene silencing}} in Hereditary transthyretin-mediated amyloidosis
Published	10/19/2024	Describe the key differences between non-homologous end joining (NHEJ) and homology directed repair (HDR) in CRISPR.
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