Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Cell Specific Gene Expression01:58

Cell Specific Gene Expression

17.5K
Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
17.5K
Transgenic Plants02:50

Transgenic Plants

9.3K
Recombinant DNA technology called transgenesis is often used to add a foreign gene or remove a detrimental gene from an organism. Such genetically modified organisms are called transgenic organisms.
The first-ever transgenic plant was a tobacco plant developed in 1983 that showed resistance against the tobacco mosaic virus. Since then, many transgenic plants have been developed and commercialized for improving the agricultural, ornamental, and horticultural value of a crop plant. Transgenic...
9.3K
Production of Pharmaceuticals01:30

Production of Pharmaceuticals

77
Industrial insulin production uses genetically engineered E. coli expressing a proinsulin gene controlled by a tryptophan promoter and containing a methionine linker for later cleavage. The cells also carry ampicillin resistance for selective growth. Seed cultures are stored at −80 °C and production begins by thawing a small amount to inoculate starter cultures, which are progressively scaled to a 50,000-L bioreactor. In the bioreactor, E. coli grow in nutrient-rich media under...
77

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Author Correction: Phage therapy with nebulized cocktail BX004-A for chronic Pseudomonas aeruginosa infections in cystic fibrosis: a randomized first-in-human trial.

Nature communications·2026
Same author

Phage therapy with nebulized cocktail BX004-A for chronic Pseudomonas aeruginosa infections in cystic fibrosis: a randomized first-in-human trial.

Nature communications·2025
Same author

Challenges in Gaucher disease: Perspectives from an expert panel.

Molecular genetics and metabolism·2025
Same author

Intrinsic link between PGRN and Gba1 D409V mutation dosage in potentiating Gaucher disease.

Human molecular genetics·2024
Same author

Bacteriophage therapy against pathological Klebsiella pneumoniae ameliorates the course of primary sclerosing cholangitis.

Nature communications·2023
Same author

PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, <i>GBA1</i> mutation-associated pathologies and diseases.

Proceedings of the National Academy of Sciences of the United States of America·2022

Related Experiment Video

Updated: Apr 18, 2026

Expression of Recombinant Cellulase Cel5A from Trichoderma reesei in Tobacco Plants
13:05

Expression of Recombinant Cellulase Cel5A from Trichoderma reesei in Tobacco Plants

Published on: June 13, 2014

13.4K

Taliglucerase alfa: an enzyme replacement therapy using plant cell expression technology.

Gregory A Grabowski1, Myriam Golembo2, Yoseph Shaaltiel2

  • 1Division of Human Genetics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

Molecular Genetics and Metabolism
|March 18, 2014
PubMed
Summary

Taliglucerase alfa, a plant-derived enzyme replacement therapy (ERT), effectively treats Gaucher disease type 1 (GD1) by improving organ and blood cell issues. This ERT offers a cost-effective alternative without requiring additional glycosylation modifications.

Keywords:
Acid β-glucosidaseGaucher diseaseInborn errors of metabolismLysosomal storage disease

More Related Videos

A Comparative Analysis of Recombinant Protein Expression in Different Biofactories: Bacteria, Insect Cells and Plant Systems
09:11

A Comparative Analysis of Recombinant Protein Expression in Different Biofactories: Bacteria, Insect Cells and Plant Systems

Published on: March 23, 2015

24.8K
In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

8.7K

Related Experiment Videos

Last Updated: Apr 18, 2026

Expression of Recombinant Cellulase Cel5A from Trichoderma reesei in Tobacco Plants
13:05

Expression of Recombinant Cellulase Cel5A from Trichoderma reesei in Tobacco Plants

Published on: June 13, 2014

13.4K
A Comparative Analysis of Recombinant Protein Expression in Different Biofactories: Bacteria, Insect Cells and Plant Systems
09:11

A Comparative Analysis of Recombinant Protein Expression in Different Biofactories: Bacteria, Insect Cells and Plant Systems

Published on: March 23, 2015

24.8K
In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

8.7K

Area of Science:

  • Biochemistry and Genetic Disorders
  • Enzyme Replacement Therapy (ERT)
  • Lysosomal Storage Disorders

Background:

  • Gaucher disease (GD) is a rare genetic disorder caused by acid β-glucosidase deficiency, leading to multi-organ dysfunction.
  • Effective ERT for GD requires specific glycosylation of enzymes for macrophage uptake.
  • Current ERT options involve complex production processes and higher costs due to glycosylation modifications.

Purpose of the Study:

  • To evaluate the efficacy and safety of taliglucerase alfa, a plant cell-expressed ERT, for treating Gaucher disease type 1 (GD1).
  • To highlight the advantages of a plant-based expression system for ERT production.

Main Methods:

  • Taliglucerase alfa is produced using a plant-based expression system (carrot root cell cultures).
  • This system naturally yields enzymes with exposed mannose residues, eliminating the need for post-production glycosidic modifications.
  • Clinical trials assessed taliglucerase alfa's impact on organomegaly and hematologic parameters in adult GD1 patients.

Main Results:

  • Taliglucerase alfa demonstrated significant efficacy in reducing organomegaly and improving hematologic parameters in adult GD1 patients.
  • The therapy showed a well-established safety profile in both ERT-naïve and previously treated patients.
  • Patients achieved and maintained therapeutic benefits, with improvements noted as early as six months.

Conclusions:

  • Taliglucerase alfa is an effective and safe ERT option for adult patients with symptomatic GD1.
  • The plant-based production method offers a potentially more cost-effective approach to ERT.
  • Further trials will explore long-term outcomes in diverse GD1 patient populations.