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Gene Therapy

Gene Therapy

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 inserted. The...

Gene Therapy

Gene Therapy

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 inserted. The...

Pharmacogenomics: Identification of New Drug Targets

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...

Human Genetics

Human Genetics

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Related Experiment Video

Updated: May 17, 2026

Candidate Gene Testing in Clinical Cohort Studies with Multiplexed Genotyping and Mass Spectrometry

Candidate Gene Testing in Clinical Cohort Studies with Multiplexed Genotyping and Mass Spectrometry

Published on: June 21, 2018

Selecting Candidates for Genetic Therapy: A Patient-Centered Framework.

Marc Henein¹, Yannis Trakadis¹

¹Division of Medical Genetics, Department of Specialized Medicine, McGill University Health Centre, Montreal, QC, Canada.

Current Gene Therapy

|May 15, 2026

Summary

Genetic therapies offer new hope for monogenic disorders but require careful patient selection. A new framework with five clinical questions aids in identifying suitable candidates for these advanced treatments.

Keywords:

Patient-centered framework aid candidate selection clinical genetic therapy genetic treatment patient prioritization tool

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Area of Science:

Genetics and Genomics
Medical Therapeutics
Bioethics

Background:

Genetic therapies are rapidly advancing for monogenic disorders, marking a significant medical breakthrough.
These novel treatments involve substantial risks and high costs, necessitating a structured approach to patient eligibility.
Current patient selection methods may not adequately address the complexities of genetic therapy.

Purpose of the Study:

To develop a systematic, patient-centered framework for selecting candidates for genetic therapy.
To establish clear clinical criteria for evaluating suitability for advanced genetic treatments.
To provide a practical tool for clinicians navigating the complexities of genetic therapy implementation.

Main Methods:

Development of a framework based on five key clinical questions.
The framework focuses on a patient-centered approach to eligibility assessment.
Application of the framework demonstrated through illustrative case examples.

Main Results:

The proposed framework offers a systematic method for genetic therapy patient selection.
Illustrative examples demonstrate the practical utility of the five-question framework.
The framework facilitates informed decision-making in the context of novel genetic treatments.

Conclusions:

A structured, patient-centered framework is essential for optimizing genetic therapy candidate selection.
The proposed five-question approach provides a valuable tool for clinicians.
Systematic patient selection is crucial for maximizing the benefits and minimizing risks of genetic therapies.