20240009284. METHOD AND COMPOSITION FOR TREATING ARGINASE 1 DEFICIENCY simplified abstract (Aerase, Inc.)

METHOD AND COMPOSITION FOR TREATING ARGINASE 1 DEFICIENCY

Organization Name

Aerase, Inc.

Inventor(s)

Scott W. Rowlinson of Austin TX (US)

Anthony G. Quinn of Gloucester MA (US)

Ann Lowe of Austin TX (US)

David Lowe of Austin TX (US)

METHOD AND COMPOSITION FOR TREATING ARGINASE 1 DEFICIENCY - A simplified explanation of the abstract

This abstract first appeared for US patent application 20240009284 titled 'METHOD AND COMPOSITION FOR TREATING ARGINASE 1 DEFICIENCY

Simplified Explanation

The abstract of the patent application describes a method and composition for treating individuals with arginase 1 deficiency (arg1-d) by rapidly reducing the levels of arginine and/or guanidino compounds in their bodies.

• The patent application proposes a method and composition to treat individuals with arg1-d.
• The treatment aims to rapidly reduce the levels of arginine and/or guanidino compounds in the subject.
• The method and composition may involve the use of specific drugs or compounds.
• The invention provides a solution for individuals suffering from arg1-d by addressing the abnormal levels of arginine and guanidino compounds in their bodies.

Potential Applications:

• Treatment of individuals with arginase 1 deficiency (arg1-d).
• Management of symptoms associated with arg1-d.
• Potential use in gene therapy or genetic engineering to address arg1-d.

Problems Solved:

• Arginase 1 deficiency (arg1-d) is a rare genetic disorder that leads to the accumulation of arginine and guanidino compounds in the body.
• The patent application proposes a method to rapidly reduce the levels of these compounds in individuals with arg1-d.
• This technology addresses the metabolic imbalance caused by arg1-d.

Benefits:

• Provides a potential treatment option for individuals with arginase 1 deficiency (arg1-d).
• Rapidly reduces the levels of arginine and/or guanidino compounds in the subject's body.
• May alleviate symptoms associated with arg1-d and improve the overall quality of life for affected individuals.

Original Abstract Submitted

a method and composition to treat a subject with arginase 1 (arg1) deficiency (arg1-d) and to rapidly reduce the levels of at least one of arginine and/or a guanidino compound in the subject.