Mammalian Expression Vector Signal Sequence Optimization

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Solution Overview

Problem

Current methods for producing recombinant proteins in mammalian cells, such as CHO cells, face challenges in efficiently secreting non-secretion competent polypeptides, like antibody chains, due to the unpredictability of signal sequences across species, leading to moderate expression levels and complex purification processes.

Innovation Solution

An expression cassette is developed with a promoter linked to a DNA sequence encoding a signal peptide, specifically selected from sequences like SEQ ID No. 10 or 9, to facilitate efficient secretion of heterologous proteins in mammalian host cells, including CHO cells, by targeting the secretory pathway and ensuring proper processing.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Productivity

If native signal sequences are used for recombinant protein secretion in mammalian cells, then the protein can be targeted to the secretory pathway, but secretion efficiency is low and unpredictable due to species-specific incompatibility

Engineering Contradiction:
Improvesecretion efficiencyVSAvoidsecretion reliability
Core Design Contradiction:
ProductivityVSReliability

Solution Approach 1:

The patent changes the amino acid sequence parameters of the signal peptide to create a human-optimized sequence (V19) that is compatible with mammalian cell secretory pathways. By modifying the signal peptide sequence while maintaining its functional properties, the invention achieves reliable and efficient secretion of recombinant proteins in mammalian cells, resolving the species-specific incompatibility issue.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent introduces a human-optimized signal peptide V19 as an intermediary element that mediates between the heterologous gene and the mammalian cell secretory system. This optimized signal peptide acts as a bridge that enables efficient recognition and processing by the human secretory pathway, improving both secretion efficiency and reliability.

Inventive Principle:
Principle #24Intermediary (Mediator)

2Productivity

If intracellular proteins are expressed at high levels, then protein production increases, but purification becomes complex requiring cell lysis and multiple isolation steps

Engineering Contradiction:
Improveprotein production levelVSAvoidpurification process complexity
Core Design Contradiction:
ProductivityVSDevice complexity

Solution Approach 1:

The patent extracts the protein from the intracellular compartment and directs it to the secretory pathway for extracellular release. By using the optimized signal peptide V19, the recombinant protein is secreted into the culture medium, separating it from the complex intracellular environment and enabling simpler purification processes that avoid cell lysis and multiple isolation steps.

Inventive Principle:
Principle #2Taking out (Extraction)

Solution Approach 2:

The patent employs the cell's own secretory machinery to automatically export the recombinant protein to the extracellular medium. The optimized signal peptide V19 directs the protein through the endoplasmic reticulum and Golgi apparatus, utilizing the cell's inherent secretion capabilities to deliver the protein externally, thereby simplifying downstream purification.

Inventive Principle:
Principle #25Self-service

3Ease of operation

If secretion is achieved using conventional signal sequences, then protein can be released into culture medium, but secretion function is insufficient due to poor operability of native signal sequences in host cells

Engineering Contradiction:
Improvesecretion functionalityVSAvoidsecretion level
Core Design Contradiction:
Ease of operationVSProductivity

Solution Approach 1:

The patent optimizes the signal peptide sequence parameters to create V19, which has improved amino acid composition and structural properties suitable for human cell recognition and processing. This parameter optimization enhances both the functionality and efficiency of protein secretion in mammalian cells.

Inventive Principle:
Principle #35Parameter changes

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

This approach results in improved secretion and increased mean antibody concentrations, with signal sequence V19 showing a particularly strong increase, demonstrating enhanced protein production and simplifying purification by secreting proteins into the extracellular medium.

Implementation Method 1

This 'preprotein' form includes an additional peptide sequence at the amino-terminus which is required to target the nascent peptide chain to the endoplasmatic reticulum to enable its entry into the secretory pathway

Methodology Applied
Scientific EffectSignal sequence-mediated targeting:

Implementation Method 2

Successful protein secretion requires effective translocation of the protein across the endoplasmatic reticulum or plasma membrane

Methodology Applied
Scientific EffectSecretory pathway translocation:

Data Source

PatentEP2152728B1Mammalian expression vector with a highly efficient secretory signal sequence
Publication Date: 2013.03.13 LONZA BIOLOGICS PLC
  • EP2152728B1 patent drawingFigure 1
  • EP2152728B1 patent drawingFigure 2
  • EP2152728B1 patent drawingFigure 3

AI summary

The present invention relates to a mammalian cell based expression and secretion system and the expression and secretion of recombinant proteins by using secretory signal peptides. The present invention also relates to an expression cassette useful for the secretion of a heterologous gene from a mammalian cell, in particular a CHO cell. The present invention is also directed to a method of secreting a heterologous protein from mammalian cells such as CHO cells.