Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

Lysosomal Hydrolases

Lysosomal Hydrolases

Lysosomes are the site for the degradation of macromolecules and biological polymers released during membrane trafficking events such as secretory, endocytic, autophagic, and phagocytic pathways. The membrane-enclosed area of the lysosome, called the lumen, contains hydrolytic enzymes active in an acidic environment. These acid hydrolases are functional at a pH between 4.5 and 5 and are involved in cellular processes such as cell signaling, energy metabolism, restoration of the plasma membrane,...

Lysosomes

Lysosomes

Lysosomes are membrane-enclosed spherical sacs derived from the Golgi apparatus. The most important function of the lysosome is degrading macromolecules and biological polymers that are released during membrane trafficking events such as the secretory, endocytic, autophagic, and phagocytic pathways. The degradation is carried out by several hydrolytic enzymes active in an acidic environment of the lysosomal lumen. These acid hydrolases are involved in cellular processes such as cell signaling,...

Lysosomes

Lysosomes

Lysosomes are membrane-enclosed spherical sacs derived from the Golgi apparatus. The most important function of the lysosome is degrading macromolecules and biological polymers that are released during membrane trafficking events such as the secretory, endocytic, autophagic, and phagocytic pathways. The degradation is carried out by several hydrolytic enzymes active in an acidic environment of the lysosomal lumen. These acid hydrolases are involved in cellular processes such as cell signaling,...

Delivery Pathways to the Lysosome

Delivery Pathways to the Lysosome

Eukaryotic cells use different mechanisms to eliminate toxic waste obsolete and worn-out substances. Lysosomes play a pivotal role in this, and hence, these substances are carried to the lysosome from other parts of the cell and extracellular space through different pathways. The most elaborately studied pathways to the lysosome are the endocytic pathways.
Endocytosis
In endocytosis, the cell membrane takes up macromolecules and particles from the surrounding medium. Clathrin-mediated...

Cystic Fibrosis: Management

Cystic Fibrosis: Management

Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
Sinus disease and chronic sinusitis...

Nephrotic Syndrome III : Nursing Management

Nephrotic Syndrome III : Nursing Management

Nursing management for nephrotic syndrome adapts as the disease progresses, with strategies evolving to address advancing symptoms and complications.Early-Stage Management In the early stages, nursing interventions for nephrotic syndrome resemble those used in managing acute glomerulonephritis, focusing on symptom monitoring, fluid balance, and managing mild to moderate edema.Vital Signs: Regularly monitor blood pressure, pulse, respiratory rate, and temperature to promptly identify...

You might also read

Related Articles

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

Sort by

Same author

Flexible Bronchoscopy in the Intensive Care Unit: Controversies, Clinical Applications, and the Expanding Role of Intensivists.

Journal of clinical medicine·2026

Same author

Non-Anastomotic Bronchial Stenosis Leading to Dual Lobar Collapse and Ex Vacuo Pleural Effusion: A Case Report.

Transplantation proceedings·2026

Same author

Endobronchial Ultrasound-guided Transbronchial Needle Aspiration Detection of PET-CT Negative Contralateral Hilar Lymph Node Metastasis in the Staging of Non-small cell Lung Cancer.

Journal of bronchology & interventional pulmonology·2026

Same author

Allograft and recipient tissue injury during cardiac allograft rejection: Evidence from cell-free DNA.

American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons·2026

Same author

Interstitial Lung Disease in 2025-Progress, Challenges, and Hope Ahead.

Journal of clinical medicine·2025

Same author

Kaposi Sarcoma in Two Lung Transplant Recipients: A Single-Center Experience.

Transplantation proceedings·2024

Search research articles

Related Experiment Video

Updated: Jul 6, 2026

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

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

Published on: December 20, 2017

Home therapy for lysosomal storage disorders.

Derryalynn A Hughes¹, Alan Mlilligan, Atul Mehta

¹Department of Academic Haematology, Royal Free Hospital and University College Medical School, London, UK.

British Journal of Nursing (Mark Allen Publishing)

|March 26, 2008

Summary

Home enzyme replacement therapy (ERT) for lysosomal storage disorders (LSDs) offers a convenient alternative to hospital visits. This approach improves patient quality of life and reduces healthcare system burden.

More Related Videos

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics

Published on: June 23, 2022

Microfluidic Production of Lysolipid-Containing Temperature-Sensitive Liposomes

Microfluidic Production of Lysolipid-Containing Temperature-Sensitive Liposomes

Published on: March 3, 2020

Related Experiment Videos

Last Updated: Jul 6, 2026

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

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

Published on: December 20, 2017

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics

Published on: June 23, 2022

Microfluidic Production of Lysolipid-Containing Temperature-Sensitive Liposomes

Microfluidic Production of Lysolipid-Containing Temperature-Sensitive Liposomes

Published on: March 3, 2020

Area of Science:

Biochemistry
Genetics
Pharmacology

Background:

Lysosomal storage disorders (LSDs) result from enzyme deficiencies, causing substrate accumulation.
Intravenous enzyme replacement therapy (ERT) is a key treatment for LSDs.
Current ERT requires frequent, often burdensome, hospital visits.

Purpose of the Study:

To evaluate the feasibility and benefits of home-based ERT for LSDs.
To assess patient compliance and quality of life with home ERT.

Main Methods:

Review of existing literature and clinical practices for home ERT.
Analysis of patient feedback and compliance data from home infusion settings.

Main Results:

Home ERT administration reduces patient travel time and hospital resource utilization.
Patients report improved independence and quality of life with home-based therapy.
High compliance rates observed for home infusions, supported by a robust community infrastructure.

Conclusions:

Home-based ERT is a viable and beneficial treatment modality for LSDs.
This approach enhances patient autonomy and potentially improves long-term outcomes.
Successful implementation requires organized community support and safety protocols.