# How Oligonucleotides Target C9orf72 for Brain Diseases > This patent describes using special genetic molecules called oligonucleotides to treat brain diseases like ALS and FTD by targeting a specific gene called C9orf72. - **Patent:** US 12735703 - **Original title:** Oligonucleotide compositions and methods of use thereof - **Granted:** 2026 - **Status:** Active - **Times cited:** 0 - **Field:** biotech, pharmaceutical, gene_editing ## What it does The patent describes specific oligonucleotide compositions designed to interact with the C9orf72 gene. These oligonucleotides are intended to be used in methods for treating conditions linked to this gene, such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). For example, a patient with C9orf72-associated ALS could receive a composition containing these oligonucleotides to potentially reduce the harmful effects of the gene. ## What it does NOT cover - Does not cover treatments for ALS or FTD that are not linked to the C9orf72 gene mutation. - Does not cover therapies for C9orf72-associated conditions that use non-oligonucleotide approaches, such as small molecule drugs or gene editing tools like CRISPR. - Does not cover diagnostic methods for identifying the C9orf72 mutation. - Does not cover oligonucleotides designed to target genes other than C9orf72. ## The clever bit The clever bit is precisely designing oligonucleotide molecules to specifically interfere with the C9orf72 gene, which is known to cause certain forms of ALS and FTD. This targeted approach aims to correct or mitigate the genetic defect rather than just treating symptoms. ## Real-world examples 1. Investigational oligonucleotide therapies for C9orf72-ALS 2. Antisense oligonucleotide drugs in development 3. Gene-targeted treatments for neurodegenerative disorders ## Why it matters This patent addresses severe neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which currently have limited treatment options. The C9orf72 gene mutation is a major genetic cause for both conditions, making targeted therapies crucial. Developing specific oligonucleotides to modulate this gene could offer a new therapeutic approach for patients suffering from these debilitating diseases. ## Frequently asked questions ### What does How Oligonucleotides Target C9orf72 for Brain Diseases cover? This patent describes using special genetic molecules called oligonucleotides to treat brain diseases like ALS and FTD by targeting a specific gene called C9orf72. ### When does this patent expire? This patent is expected to expire on September 15, 2046, when the invention enters the public domain. ### What problem does this patent solve? This patent addresses severe neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which currently have limited treatment options. The C9orf72 gene mutation is a major genetic cause for both conditions, making targeted therapies crucial. Developing specific oligonucleotides to modulate this gene could offer a new therapeutic approach for patients suffering from these debilitating diseases. ### What does this patent NOT cover? Does not cover treatments for ALS or FTD that are not linked to the C9orf72 gene mutation. **Full plain-English explainer:** https://patentbrief.org/patent/us/12735703/oligonucleotide-compositions-and-methods-of-use-thereof **Original patent:** https://patents.google.com/patent/US12735703 --- _Source: PatentBrief — https://patentbrief.org. Patent facts are from public records; the plain-English explanation is PatentBrief's._ ## Related patents Semantically similar inventions in the PatentBrief corpus: - [How to Design Effective siRNA Molecules to Silence Specific Genes](https://patentbrief.org/patent/us/9839649/methods-and-compositions-for-selecting-sirna-of-improved-functionality) — A patent detailing the precise chemical structure and sequence requirements for siRNA molecules designed to effectively turn off the AAT gene in cells. - [Using Synthetic DNA Fragments to Block HIV Replication](https://patentbrief.org/patent/us/5110802/crixivan-indinavir) — A 1987 patent describing a specific synthetic DNA molecule designed to stop the HIV virus from replicating by blocking a key part of its genetic code. - [Diagnosing Brain Conditions by Watching Lab-Grown Neurons Light Up](https://patentbrief.org/patent/us/12736528/diagnostic-methods-for-neural-disorders) — This patent describes a method to diagnose brain disorders by taking a patient's cell, turning it into a neuron, making it glow when active, and comparing its light patterns to a genetically matched healthy cell. - [How Modified RNA Tricks Cells Into Making Proteins Without Triggering Attacks](https://patentbrief.org/patent/us/8278036/rna-containing-modified-nucleosides-and-methods-of-use-thereof) — A breakthrough method for using modified RNA to deliver instructions to cells without causing the body to reject the treatment as a foreign invader. - [How CRISPR-Cas9 Uses RNA to Edit DNA](https://patentbrief.org/patent/us/10113167/methods-and-compositions-for-rna-directed-target-dna-modification-and-for-rna-directed-modulation-of-transcription) — This patent describes the fundamental mechanism of using a two-part RNA system to guide the Cas9 protein to specific locations in DNA for precise editing.