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Strategies targeting mRNA: RNA-based drugs (RAS Protein (Ras Gene (H-RAS,…
Strategies targeting mRNA: RNA-based drugs
RNA vs si/miRNA (Fig 55)
Functions
Produce Required Proteins only
Regulate Protein Types
Separate bone/skin cells
Matches protein sequence that wants removal
Coding Genes: 20%
Non-Coding Genes: 80%
Does not make proteins
Comparisons
Difference
miRNA
Partial Paired
Diagnostic Tool
siRNA
Completely Paired
Can only knock down specific thing @ 1 time
RAS Protein
Ras Gene
H-RAS
C-RAS
K-RAS
N-RAS
Localised to inner surface of cell membrane
Mutation
Tumor
Cancer
Cure
Gene Inhibition Therapy
siRNA/miRNA
mRNA knockdown
Crisper Cas9 Technology
mRNA editing/correcting
Antisense Technology
mRNA blocking
Exon Skipping
mRNA masking
RNAi
siRNA
Function
Knockout specific protein production with high specificity
Potential Cancer Targets (Fig 56)
Treatment Process
Diagnosis & Prognosis => Remission => Relapse => Metastasis
Applications
Therapeutic
Antisense Delivery
Combination Therapies
Therapeutic miR delivery
miRNA
Gene Correction
Exon Skipping Technology
Antisense Technology
Single Strand DNA/RNA complete match with mRNA
Works in cells & animal models
Gene Interference Technology
Gene Correction in DNA
CRISPER Cas9
Challenges
Enzymatic Stability
Solution
Chemical Modification of RNA & DNA
siRNA into drugs (Fig 57)
DNA vs RNA
DNA
No OH Group
Base T
Easy to Complex
RNA
OH Group
Base U
Difficult to Complex