Amyotrophic lateral sclerosis (ALS) is a relentless disease that eliminates muscle control and often leads to severe disability and ultimate death within a few years of diagnosis. Although traditionally the treatment focused on relieving symptoms, none successfully stopped or reversed the progression of ALS. Now, a new breakthrough is expected to change this narrative by targeting the genetic roots of the disease. Using sophisticated gene silencing techniques, the researchers developed a therapy that not only extends lifespan but also significantly improves motor function in the ALS model, thus bringing a glimmer of hope to those affected by this devastating condition.
Leaded by Dr. Longchen Li and Dr. Robert Place of Ractigen Therapeutics, along with their colleagues Chunling Duan, Moorim Kang, Xiaojie Pan, Dr. Zubao Gan, Dr. Vera Huang, Dr. Vera Huang, and Ractigen T Guanlin Li of herapeutics has developed a The desired gene recursion therapy can extend lifespan and improve motor function in a mouse model of amyotrophic lateral sclerosis (ALS), a severe neurodegenerative disease. The study focuses on a novel small interfering RNA (siRNA), showing great potential to advance ALS treatment. Recently published in the journal Molecular Therapy: Nucleic Acids, the study marks an important milestone in the fight against ALS.
“This siRNA-ACO represents a novel way to deliver double-stranded RNA into the central nervous system and is currently undergoing ALS treatment in clinics,” said Dr. Li, a key challenge in treating neurological diseases.
The method shows that this new siRNA-ACO compound can be administered by intrathecal injection to reach the central nervous system, where it remains active for up to eight weeks. This lasting effect is crucial for ALS, and the disease progresses rapidly after diagnosis. The treated mice not only lived longer, but also showed better motor function compared to the mice that received previously treated.
“Intraventricular and intrathecal administration delays disease progression and expands animal survival in a dose-dependent manner,” Dr. Lee explained.
In addition, the ability of this therapy to prevent motor loss is particularly noteworthy. “Treatment can also prevent degradation of motor function associated with the disease, including improving animal migration, muscle strength and coordination,” added Dr. Lee. These improvements are a key sign of the potential of the therapy to significantly improve the quality of life in patients with ALS.
The implications of this study are huge, providing hope for this new treatment to provide relief soon for patients with ALS. By directly targeting the genetic basis of ALS, this approach not only hopes to slow down but also potentially stop or reverse muscle degeneration representing the disease, thereby significantly improving patient outcomes.
Dr. Li and colleagues are committed to further refine this treatment and plan to initiate clinical trials that can establish new areas of gene therapy in ALS and other neurodegenerative diseases. With the attention of the scientific and medical community, this study represents an important step in seeking to understand and fight one of the most challenging diseases of the nervous system.
Journal Reference
Chunling Duan, Moorim Kang, Xiaojie Pan, etc. “Intrathecal administration of novel siRNA modalities expands survival and improves motor function in the SOD1G93A ALS mouse model.” Molecular Therapy: Nucleic Acid (2024): doi:
