# How to Design Effective siRNA Molecules to Silence Specific Genes

> A patent detailing the precise chemical structure and sequence requirements for siRNA molecules designed to effectively turn off the AAT gene in cells.

- **Patent:** US 9839649
- **Original title:** Methods and compositions for selecting siRNA of improved functionality
- **Owner:** Thermo Fisher Scientific Inc
- **Granted:** 2017
- **Status:** Active
- **Times cited:** 3
- **Field:** biotech, pharmaceutical

## What it does

This patent describes the structural requirements for small interfering RNA (siRNA) molecules used to trigger gene silencing. It specifies that the molecule must consist of a sense and antisense strand, each between 19 and 30 nucleotides long, forming a duplex region. Crucially, the antisense strand must be a complement to a specific target sequence identified as SEQ ID NO:465, which corresponds to the AAT gene. The patent allows for variations in the presence or absence of short nucleotide overhangs at the ends of the duplex.

## What it does NOT cover

- Does not cover siRNA molecules that target genes other than the AAT gene specified by SEQ ID NO:465.
- Does not cover RNA molecules longer than 30 nucleotides.
- Does not cover single-stranded RNA molecules.
- Does not cover methods of delivering these molecules into a living organism.

## The clever bit

The innovation lies in the rational design criteria: by strictly defining the length (19-30 nucleotides) and the exact sequence complement required for the antisense strand, the inventors ensure the siRNA molecule is optimized for the cell's natural silencing machinery, minimizing off-target effects.

## Real-world examples

1. RNA interference research tools
2. Experimental gene silencing therapies for Alpha-1 Antitrypsin deficiency

## Why it matters

This patent is significant for the field of RNA interference (RNAi), a biological process where RNA molecules inhibit gene expression. By defining the exact sequence and structural parameters for targeting the AAT gene—often associated with Alpha-1 Antitrypsin deficiency—it provides a blueprint for developing therapeutic reagents that can precisely 'switch off' disease-causing genes.

## Frequently asked questions

### What does How to Design Effective siRNA Molecules to Silence Specific Genes cover?

A patent detailing the precise chemical structure and sequence requirements for siRNA molecules designed to effectively turn off the AAT gene in cells.

### Who owns patent US 9839649?

Thermo Fisher Scientific Inc owns this patent, granted in 2017.

### When does this patent expire?

This patent is expected to expire on November 9, 2035, when the invention enters the public domain.

### What is patent US 9839649 cited by?

This patent has been cited by 3 later patents that build on its ideas.

### What problem does this patent solve?

This patent is significant for the field of RNA interference (RNAi), a biological process where RNA molecules inhibit gene expression. By defining the exact sequence and structural parameters for targeting the AAT gene—often associated with Alpha-1 Antitrypsin deficiency—it provides a blueprint for developing therapeutic reagents that can precisely 'switch off' disease-causing genes.

### What does this patent NOT cover?

Does not cover siRNA molecules that target genes other than the AAT gene specified by SEQ ID NO:465.

**Full plain-English explainer:** https://patentbrief.org/patent/us/9839649/methods-and-compositions-for-selecting-sirna-of-improved-functionality

**Original patent:** https://patents.google.com/patent/US9839649

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_Source: PatentBrief — https://patentbrief.org. Patent facts are from public records; the plain-English explanation is PatentBrief's._


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