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Ethical Cell and Gene Therapy & Regenerative Medicine - Healing Genes
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Ethical Cell and Gene Therapy & Regenerative Medicine

We are committed to working diligently to bring you accurate, up-to-date news, information, and education about ethical cell and gene therapy as well as regenerative medicine.

We also are committed to ensuring you have up-to-date information on gene medicine discussions and trends, including some of the more controversial topics.

To accomplish our goals of sharing accurate information with transparency, we believe it’s important to share where we stand on the bioethics of cell and gene therapy today.

Genome Editing

Genome editing of somatic, non-germline/”adult” cells, leads to changes that are not passed to subsequent generations. Most people, including most religious leaders who have discussed the ethics of genome editing, appear to support the ethical, therapeutic use of genome editing technologies of somatic cells.

You will find many official position papers here.

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On the other hand, germline genome-editing techniques, where egg and sperm cells are treated, have not advanced to the point where human trials would be appropriate. As the science evolves, the public, public policy and science leaders say numerous criteria regarding the safety, legality, and ethics of germline genome editing must be met before human trials should begin.

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Stem Cells in Medicine

Stem cell therapy, a treatment that uses the body’s special rejuvenating cells to replace or repair damaged cells or tissues, shows promise and has been used successfully in a few applications, such as bone marrow transplants (sickle cell disease); beta-thalassemia; some bone, skin, and eye injuries/diseases; and certain blood cancers. The marketing of unproven stem cell treatments is growing, and this retail marketing of such serious science raises major ethical concerns. Unapproved treatments have been marketed as cutting-edge therapy, but may not have been rigorously tested in approved clinical trials. They may be unsafe or ineffective.

Why wouldn’t people be in favor of germline editing? If we alter genes in sperm, eggs, or embryos, those changes will be passed down to future generations. Unintended, negative effects may not become apparent for years. Moreover, the babies who will receive those edited genes will have no opportunity to evaluate or agree to those changes.

People support and encourage the continued efforts to engage stakeholder groups from around the world — including patients, scientists, bioethicists, regulatory authorities, theologians, and various international governing bodies — in the dialogue about the ethical and legal implications of genome editing.

We are committed to providing scientifically-grounded education on this topic.

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Somatic cells account for the majority of cells in the body. Most genetic diseases can be treated in somatic cells. The DNA in these cells is non-heritable, which means that the editing modifications would affect only the patient and not be transmitted to the patient’s children.

Germline cells are those involved in reproduction (sperm or egg cells). Because of this, genome editing approaches that target germline cells permanently change the DNA.

Cultural Sensitivity

Policy & Regulations

Policy & Regulations

New FDA Model Aims to Speed More Rare Disease Treatments to Patients

The development of new therapies to treat orphan diseases is particularly perilous, due to limited patient populations, high costs, and difficulty in measuring clinical effectiveness. FDA officials are addressing these issues through the Rare Disease Cures Accelerator (RDCA) initiative in the Center for Drug Evaluation …

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Policy & Regulations

New International Commission Launched on Clinical Use of Heritable Human Genome Editing

An international commission has been convened by the U.S. National Academy of Medicine (NAM), the U.S. National Academy of Sciences (NAS), and the Royal Society of the U.K., with the participation of science and medical academies around the world, to develop a framework for scientists, …

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Policy & Regulations

FDA’s Efforts to Advance the Development of Gene Therapy

Gene therapy has been on the horizon for several decades and has now become a reality in the United States. There are now three approved products: two cell-based gene therapies for cancers of the blood (Kymriah and Yescarta) and one directly-administered gene therapy (Luxturna) for …

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