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Related Concept Videos

Gene Therapy00:59

Gene Therapy

27.7K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Group Therapy01:26

Group Therapy

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Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
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Gene Flow02:39

Gene Flow

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Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
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Gene Families01:57

Gene Families

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Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
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Gene Conversion02:08

Gene Conversion

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Other than maintaining genome stability via DNA repair, homologous recombination plays an important role in diversifying the genome. In fact, the recombination of sequences forms the molecular basis of genomic evolution. Random and non-random permutations of genomic sequences create a library of new amalgamated sequences. These newly formed genomes can determine the fitness and survival of cells. In bacteria, homologous and non-homologous types of recombination lead to the evolution of new...
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What is Gene Expression?01:42

What is Gene Expression?

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Overview
Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
Genetic Information Flows from DNA to RNA to Protein
A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is made up of nucleotides and proteins consist of amino...
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Updated: Feb 16, 2026

Production and Purification of Baculovirus for Gene Therapy Application
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Gene therapy for Mucopolysaccharidoses.

Kazuki Sawamoto1, Hui-Hsuan Chen2, Carlos J Alméciga-Díaz3

  • 1Nemours/Alfred I. duPont Hospital for Children, Wilmington, DE, United States.

Molecular Genetics and Metabolism
|January 4, 2018
PubMed
Summary

Gene therapy offers a potential one-time cure for Mucopolysaccharidoses (MPS), a group of genetic lysosomal storage disorders. Current research focuses on advancing preclinical and clinical trials for this permanent treatment approach.

Keywords:
Adeno-associated virusGene therapyGlycosaminoglycansMucopolysaccharidoses

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Area of Science:

  • Biochemistry
  • Genetics
  • Pediatrics

Background:

  • Mucopolysaccharidoses (MPS) are inherited lysosomal storage disorders (LSDs) resulting from enzyme deficiencies.
  • Current treatments like ERT, HSCT, and SRT manage symptoms but do not offer a cure.
  • MPS disorders manifest with diverse clinical symptoms based on type and severity.

Purpose of the Study:

  • To review the development of gene therapy for MPS.
  • To summarize preclinical and clinical trial progress in gene therapy for MPS.

Main Methods:

  • Review of preclinical studies spanning three decades.
  • Analysis of ongoing clinical trials for various MPS types.

Main Results:

  • Gene therapy aims to provide a permanent, one-time treatment by correcting the underlying enzyme deficiency.
  • Significant progress has been made in preclinical gene therapy research for MPS.
  • Clinical trials are actively underway in multiple countries, including the US, Australia, and European nations.

Conclusions:

  • Gene therapy represents a promising curative strategy for MPS.
  • Ongoing clinical trials are crucial for evaluating the safety and efficacy of gene therapy in patients.
  • Further research and development are essential to establish gene therapy as a standard treatment for MPS.