Related Experiment Video

Updated: Feb 3, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
05:09

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats

Published on: May 16, 2025

3.3K

Compassionate use of gene therapies in pediatrics: An ethical analysis

Carolyn P Neuhaus1, Rachel L Zacharias1

  • 1The Hastings Center, 21 Malcolm Gordon Road, Garrison, NY 10524, United States.

Seminars in Perinatology
|November 4, 2018
PubMed

Insights

Compassionate use of CRISPR gene therapies for children raises ethical concerns. This commentary discusses impacts on research, therapy hype, and parental rights, urging careful consideration before pursuing these treatments outside clinical trials.

Area of Science:

  • Bioethics
  • Gene Therapy
  • Pediatric Medicine

Background:

  • Compassionate use programs have provided gene therapies to pediatric patients.
  • Federal Right to Try laws may increase demand for investigational gene therapies outside clinical trials.
  • Clinicians, parents, and companies require guidance on using CRISPR gene therapies compassionately for children.

Purpose of the Study:

  • To raise concerns regarding the compassionate use of CRISPR-mediated gene therapies in pediatric and perinatal populations.
  • To outline key considerations for parents, clinicians, and drug developers when evaluating compassionate use of these novel therapies for children.

Main Methods:

  • Commentary based on ethical considerations and existing precedents.
  • Analysis of potential impacts on scientific research and patient welfare.
  • Discussion of parental authority in medical decision-making for investigational treatments.

Main Results:

  • Identifies three critical issues surrounding compassionate use of CRISPR gene therapies in pediatric patients: effects on scientific research, the balance of hype versus harm, and the boundaries of parental decision-making authority.
  • Highlights the need for support systems for all stakeholders involved in these complex decisions.

Conclusions:

  • The compassionate use of CRISPR gene therapies in pediatric patients necessitates careful ethical deliberation.
  • A balanced approach is required to weigh potential benefits against risks, research integrity, and parental rights.
  • Further discussion and guidelines are needed to support informed decision-making in this evolving area of medicine.

Keywords:
Compassionate UsePediatric EthicsResearch EthicsRight to Try

More Related Videos

Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart06:07

Large Animal Model for Evaluating the Efficacy of the Gene Therapy in Ischemic Heart

Published on: September 2, 2021

2.3K
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications08:32

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications

Published on: August 9, 2022

4.4K

Related Concept Videos

Gene Therapy00:59

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...
27.6K
Ethics in Research01:56

Ethics in Research

Today, scientists agree that good research is ethical in nature and is guided by a basic respect for human dignity and safety. However, this has not always been the case. Modern researchers must demonstrate that the research they perform is ethically sound.
25.6K
Ethics and Bioethics01:22

Ethics and Bioethics

Ethics is a philosophical study of moral actions. Ethics attempts to determine what is valuable for individuals and society. It examines the rational justification of moral judgments and analyzes what is morally just, fair, and right. Bioethics is a sub-discipline of applied ethics that analyzes the philosophical, social, and legal issues in life sciences and medicine. Ethical theories serve as a foundation for decision-making and represent the viewpoints from which people seek direction. They...
2.9K
Ethical Issues01:27

Ethical Issues

Nurses are essential in patient care, upholding the ethical principles of their profession and effectively navigating ethical dilemmas. Neglecting ethical issues can lead to inadequate patient care, compromised therapeutic relationships, and moral distress among healthcare workers.
Ethical Concerns in Healthcare:
2.2K
Ethical Dilemmas II01:30

Ethical Dilemmas II

Resolving an ethical dilemma in healthcare involves a systematic approach that considers every aspect of the issue, respecting both the patient's needs and values and the healthcare professional's ethical obligations. Here are potential steps to resolve an ethical dilemma:
2.4K
Ethical Standards I01:25

Ethical Standards I

The American Nurses Association (ANA) created and implemented the first nationally accepted Code of Ethics for Nurses with Interpretive Statements. The Code of Ethics is a living document regularly updated by the ANA and establishes an ethical standard that is non-negotiable for nurses in all roles and settings.
The Code of Ethics provisions outline the nurse's duty to the patient, the healthcare team, the profession, and society. The Code's fundamental principles include advocacy,...
1.6K