18744175. COMPOSITIONS AND METHODS FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA) simplified abstract (Beam Therapeutics Inc.)
Contents
COMPOSITIONS AND METHODS FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA)
Organization Name
Inventor(s)
Shaunna Berkovitch of Cambridge MA (US)
Jason Michael Gehrke of Cambridge MA (US)
Holly Rees of Cambridge MA (US)
Conrad Rinaldi of Cambridge MA (US)
Tanggis Bohnuud of Cambridge MA (US)
COMPOSITIONS AND METHODS FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA) - A simplified explanation of the abstract
This abstract first appeared for US patent application 18744175 titled 'COMPOSITIONS AND METHODS FOR THE TREATMENT OF SPINAL MUSCULAR ATROPHY (SMA)
The invention of the disclosure features compositions and methods for treating spinal muscular atrophy (SMA) by introducing alterations to a survival of motor neuron 2 (SMN2) polynucleotide(s) in a cell. In particular embodiments, the invention provides a base editor system (e.g., a fusion protein or complex comprising a programmable DNA binding protein, a nucleobase editor, and gRNA) for modifying an SMN2 polynucleotide(s), where the alteration is associated with increased expression of the SMN2 polypeptide(s) encoded by the polynucleotide(s).
- Introduces compositions and methods for treating spinal muscular atrophy (SMA) by altering SMN2 polynucleotides in cells.
- Provides a base editor system for modifying SMN2 polynucleotides to increase expression of SMN2 polypeptides.
- Fusion protein or complex includes programmable DNA binding protein, nucleobase editor, and gRNA.
Potential Applications: - Treatment of spinal muscular atrophy (SMA). - Gene therapy for genetic disorders affecting SMN2 expression.
Problems Solved: - Lack of effective treatments for SMA. - Low expression of SMN2 polypeptides in affected individuals.
Benefits: - Improved treatment options for SMA. - Increased expression of SMN2 polypeptides for better disease management.
Commercial Applications: Title: Innovative Gene Therapy for Spinal Muscular Atrophy (SMA) Description: This technology can be utilized in the development of gene therapy treatments for SMA, catering to a niche market of patients with genetic disorders affecting SMN2 expression. The commercial implications include potential partnerships with pharmaceutical companies for product development and distribution.
Prior Art: Readers can explore prior research on gene editing technologies, base editing systems, and gene therapy approaches for SMA treatment.
Frequently Updated Research: Stay updated on advancements in gene editing techniques, base editor systems, and gene therapy applications for SMA through scientific journals and research publications.
Questions about Gene Therapy for Spinal Muscular Atrophy (SMA): 1. How does the base editor system modify SMN2 polynucleotides to increase SMN2 expression? - The base editor system utilizes a fusion protein or complex with a programmable DNA binding protein, nucleobase editor, and gRNA to introduce alterations in SMN2 polynucleotides, leading to enhanced expression of SMN2 polypeptides.
2. What are the potential long-term implications of using this gene therapy for SMA treatment? - The long-term implications may include improved disease management, better quality of life for SMA patients, and ongoing research to optimize gene therapy approaches for genetic disorders affecting SMN2 expression.
Original Abstract Submitted
The invention of the disclosure features compositions and methods for treating spinal muscular atrophy (SMA) by introducing alterations to a survival of motor neuron 2 (SMN2) polynucleotide(s) in a cell. In particular embodiments, the invention provides a base editor system (e.g., a fusion protein or complex comprising a programmable DNA binding protein, a nucleobase editor, and gRNA) for modifying an SMN2 polynucleotide(s), where the alteration is associated with increased expression of the SMN2 polypeptide(s) encoded by the polynucleotide(s).
