View source for 20240024506. POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA simplified abstract (ModernaTX, Inc.)

=POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA=


==Organization Name==

[[:Category:ModernaTX, Inc.|ModernaTX, Inc.]]


[[Category:ModernaTX, Inc.]]

==Inventor(s)==

[[:Category:Lei Jiang of Cambridge MA (US)|Lei Jiang of Cambridge MA (US)]][[Category:Lei Jiang of Cambridge MA (US)]]

[[:Category:Lin Tung Guey of Lexington MA (US)|Lin Tung Guey of Lexington MA (US)]][[Category:Lin Tung Guey of Lexington MA (US)]]

[[:Category:Paolo G. V. Martini of Boston MA (US)|Paolo G. V. Martini of Boston MA (US)]][[Category:Paolo G. V. Martini of Boston MA (US)]]

[[:Category:Vladimir Presnyak of Manchester NH (US)|Vladimir Presnyak of Manchester NH (US)]][[Category:Vladimir Presnyak of Manchester NH (US)]]

==POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA - A simplified explanation of the abstract==


This abstract first appeared for US patent application 20240024506 titled 'POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS FOR THE TREATMENT OF PROPIONIC ACIDEMIA

==Simplified Explanation==

The abstract of this patent application describes mRNA therapy for the treatment of propionic acidemia (PA). The mRNA molecules used in this invention encode human propionyl-coa carboxylase alpha (PCCA) and/or human propionyl-coa carboxylase beta (PCCB), as well as functional fragments and fusion proteins comprising PCCA and/or PCCB. These mRNA molecules are preferably encapsulated in lipid nanoparticles (LNPs) for efficient delivery to cells and tissues in subjects. The mRNA therapies increase and/or restore deficient levels of propionyl-coa carboxylase (PCC) expression and/or activity in subjects, while also decreasing levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity.

* mRNA therapy for propionic acidemia (PA)
* Encodes human propionyl-coa carboxylase alpha (PCCA) and/or human propionyl-coa carboxylase beta (PCCB)
* Functional fragments and fusion proteins of PCCA and/or PCCB
* Encapsulated in lipid nanoparticles (LNPs) for efficient delivery
* Increases and/or restores deficient levels of PCC expression and/or activity
* Decreases levels of disease-associated toxic metabolites

== Potential Applications ==
* Treatment of propionic acidemia (PA)
* Gene therapy for genetic disorders affecting propionyl-coa carboxylase (PCC) activity

== Problems Solved ==
* Deficient levels of propionyl-coa carboxylase (PCC) expression and/or activity
* Accumulation of disease-associated toxic metabolites

== Benefits ==
* Improved treatment for propionic acidemia (PA)
* Restoration of normal PCC activity
* Reduction of disease-associated toxic metabolites


==Original Abstract Submitted==

this disclosure relates to mrna therapy for the treatment of propionic acidemia (pa). mrnas for use in the invention, when administered in vivo, encode human propionyl-coa carboxylase alpha (pcca) and/or human propionyl-coa carboxylase beta (pccb), and isoforms thereof, functional fragments thereof, and fusion proteins comprising pcca and/or pccb. mrnas of the invention are preferably encapsulated in lipid nanoparticles (lnps) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mrna therapies of the invention increase and/or restore deficient levels of propionyl-coa carboxylase (pcc) expression and/or activity in subjects. mrna therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient pcca or pccb activity, in subjects.



[[Category:C12N9/00]]