Gene Silencing News and Research

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Alnylam announces positive results from Phase 3 Study of givosiran for treating acute hepatic porphyria

Alnylam announces positive results from Phase 3 Study of givosiran for treating acute hepatic porphyria

New study focuses on two-way gene switches controlling gene activity

New study focuses on two-way gene switches controlling gene activity

Antioxidant in green tea helps sneak therapeutic RNAs into cells

Antioxidant in green tea helps sneak therapeutic RNAs into cells

Penn researchers find common thread linking almost all TNR expansion diseases

Penn researchers find common thread linking almost all TNR expansion diseases

Arrowhead completes dosing of ARO-AAT Phase 1 study to treat rare genetic liver disease

Arrowhead completes dosing of ARO-AAT Phase 1 study to treat rare genetic liver disease

EC grants marketing authorization for ONPATTRO (patisiran) in Europe

EC grants marketing authorization for ONPATTRO (patisiran) in Europe

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Researchers use DNA editing tool to reveal mechanisms behind autoimmune diseases

Researchers use DNA editing tool to reveal mechanisms behind autoimmune diseases

Alnylam’s patisiran receives positive opinion from CHMP for treating hATTR amyloidosis

Alnylam’s patisiran receives positive opinion from CHMP for treating hATTR amyloidosis

Newer version of ALS drug may effectively treat inherited form of the disease

Newer version of ALS drug may effectively treat inherited form of the disease

Researchers employ nucleotide-based gene silencing to mitigate common ataxia

Researchers employ nucleotide-based gene silencing to mitigate common ataxia

Alnylam announces new positive results from Phase 1/2 study of lumasiran in patients with PH1

Alnylam announces new positive results from Phase 1/2 study of lumasiran in patients with PH1

Phase 1/2 study evaluates safety, tolerability of multiple-ascending doses of ARO-HBV in hepatitis B patients

Phase 1/2 study evaluates safety, tolerability of multiple-ascending doses of ARO-HBV in hepatitis B patients

Alnylam achieves delivery of novel siRNAs conjugates to the central nervous system

Alnylam achieves delivery of novel siRNAs conjugates to the central nervous system

Sylentis presents clinical study results of new treatment for dry eye syndrome

Sylentis presents clinical study results of new treatment for dry eye syndrome

Researchers use CRISPR/Cas9 technique to turn off gene that regulates cholesterol levels in adult mice

Researchers use CRISPR/Cas9 technique to turn off gene that regulates cholesterol levels in adult mice

Researchers target telomerase for killing NRAS-mutant melanoma cells

Researchers target telomerase for killing NRAS-mutant melanoma cells

Study reveals key function of ALS-linked protein

Study reveals key function of ALS-linked protein

New understanding of how molecules regulate enzyme could lead to potential cancer targets

New understanding of how molecules regulate enzyme could lead to potential cancer targets

Newly described super-silenced DNA reveals novel approach to reprogram cells

Newly described super-silenced DNA reveals novel approach to reprogram cells