18270014. Suppression-Replacement Gene Therapy simplified abstract (Mayo Foundation for Medical Education and Research)

Suppression-Replacement Gene Therapy

Organization Name

Mayo Foundation for Medical Education and Research

Inventor(s)

Michael J. Ackerman of Rochester MN (US)

Steven M. Dotzler of Rochester MN (US)

William Gendron of Rochester MN (US)

Sahej Bains of Rochester MN (US)

Changsung John Kim of Rochester MN (US)

David J. Tester of Rochester MN (US)

Suppression-Replacement Gene Therapy - A simplified explanation of the abstract

This abstract first appeared for US patent application 18270014 titled 'Suppression-Replacement Gene Therapy

Simplified Explanation

The abstract of the patent application describes methods and materials for treating a mammal with a congenital disease, such as congenital heart disease, by using nucleic acids to suppress expression of mutant disease-related alleles while providing replacement cDNA without the disease-related mutations.

• Methods and materials for treating a mammal with a congenital disease
• Use of nucleic acids to suppress expression of mutant disease-related alleles
• Providing replacement cDNA without disease-related mutations

Potential Applications

The technology described in the patent application could potentially be applied in the treatment of various congenital diseases in mammals, including congenital heart diseases like congenital long QT syndrome.

Problems Solved

This technology addresses the challenge of treating congenital diseases in mammals by targeting mutant disease-related alleles and providing replacement cDNA without the disease-related mutations.

Benefits

The use of nucleic acids to suppress expression of mutant disease-related alleles while providing replacement cDNA offers a targeted and potentially effective treatment approach for congenital diseases in mammals.

Potential Commercial Applications

The technology described in the patent application could have commercial applications in the development of gene therapies for congenital diseases in mammals, potentially leading to new treatment options for patients.

Possible Prior Art

One possible prior art in this field could be the use of gene editing technologies such as CRISPR-Cas9 for treating genetic diseases in mammals. Research on gene therapy for congenital heart diseases may also be relevant prior art.

What are the specific nucleic acids used in the methods described in the patent application?

The specific nucleic acids used in the methods described in the patent application are those that can suppress the expression of mutant disease-related alleles in mammals while providing replacement cDNA without the disease-related mutations.

How do the methods in the patent application compare to traditional treatments for congenital diseases in mammals?

The methods described in the patent application offer a targeted approach to treating congenital diseases in mammals by specifically addressing mutant disease-related alleles and providing replacement cDNA without the disease-related mutations. This differs from traditional treatments that may focus on symptom management rather than directly targeting the underlying genetic cause of the disease.

Original Abstract Submitted

Methods and materials for treating a mammal having a congenital disease (e.g., a congenital heart disease such as congenital long QT syndrome) are provided herein. For example, this document provides methods and materials for generating and using nucleic acids to treat a mammal having a congenital disease, where the nucleic acids can suppress expression of mutant disease-related alleles in the mammal while providing a replacement cDNA that does not contain the disease-related mutation(s).