// Evidence Grader
Claim grades, A through F
Every major genomics claim in this course gets a transparent grade — from clinically established interventions to outright misleading marketing. Use this dashboard to audit what's settled, what's preliminary, and what's hype.
A
Clinically established
B
Supported, context-specific
C
Promising, preliminary
D
Plausible, unproven
E
Popular, weak support
F
Misleading or false
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A
Inclisiran (GalNAc-siRNA against PCSK9) achieves sustained LDL-C lowering with twice-yearly dosing
ORION-9/10/11 phase 3 trials; ~50% LDL-C reduction.
Module 10 · Beyond CRISPR: ASO, siRNA, mRNA, AAV, and Cell Therapy →A
Patisiran improves polyneuropathy and quality-of-life outcomes in hereditary ATTR amyloidosis
APOLLO trial, NEJM 2018.
Module 10 · Beyond CRISPR: ASO, siRNA, mRNA, AAV, and Cell Therapy →A
Onasemnogene abeparvovec (Zolgensma) preserves motor function in pre-symptomatic SMA when given before 6 weeks
SPR1NT trial; treatment timing is decisive.
Module 10 · Beyond CRISPR: ASO, siRNA, mRNA, AAV, and Cell Therapy →B
Personalized neoantigen mRNA vaccines reduce melanoma recurrence post-resection
KEYNOTE-942 phase 2 (mRNA-4157 + pembrolizumab) HR ~0.56; phase 3 ongoing.
Module 10 · Beyond CRISPR: ASO, siRNA, mRNA, AAV, and Cell Therapy →C
CD19 CAR-T induces durable remissions in refractory autoimmune disease (SLE, myositis, systemic sclerosis)
Schett group case series and small trials are striking; no controlled phase 3 yet.
Module 10 · Beyond CRISPR: ASO, siRNA, mRNA, AAV, and Cell Therapy →F
AAV gene therapy is generally re-dosable
Pre-existing and induced neutralizing antibodies preclude conventional re-dosing for current capsids; engineered evader capsids and IgG-cleaving enzymes are research-stage.
Module 10 · Beyond CRISPR: ASO, siRNA, mRNA, AAV, and Cell Therapy →