Segments in this Video

Introduction: The Gene Doctors (01:58)


Every year more than a million babies are born with genetic diseases; gene doctors are revolutionizing medicine and therapies, giving patients hope. (Credits)

Personal Motivators (03:33)

Sonya and Eric are aspiring biomedical researchers looking for a cure to the genetic disease that inflicted her mother; with collage art, she explains the process of losing her to fatal familia insomnia, a rare illness causing neural damage. Sonya is also at risk of having the mutation.

Inherited Risks (03:22)

Genes are composed of A, T, C, and G molecules, mutation of their order can result in disease. Errors in DNA impact a cell's ability to make certain proteins, which have many different, vital health functions. Sonya gets tested for an inherited brain disorder, and results positive; she has been given until her fifties to live, and hoping to find a cure.

Correcting Vision (08:04)

Molly Troxel was born with symptoms of poor sight; she was later diagnosed with a rare inherited blindness and declared legally blind. She now sees as a result of experimental surgery; an ophthalmologist and eye surgeon first performed the operation on dogs. Viruses act as delivery vehicles for correct copy DNA. A genetic eye disease specialist explains inherited mutation, and Molly's specific condition.

Gene Therapy (04:38)

The goal of the Human Genome Project was to determine the DNA sequence of all human genes, and to revolutionize medicine. During clinical trials, Jesse Gelsinger had a severe immune response to a viral vector; he died in four days. The tragedy impacted the genetic research community, studies were shut down; in 2005, new trials were conducted with safer vectors.

Duchenne's (08:13)

Austin and Max Leclaire have been diagnosed with Duchenne Muscular Dystrophy, a rare and fatal genetic disease affecting balance and muscle function, and causing organ failure; by 20, most inflicted die of respiratory failure. A defect in the DMD gene restricts the production of dystrophin; scientists are researching a new drug allowing the protein formation; it is not a perfect solution, but is working. The clinical trials are emotionally and physically taxing; the Leclaire family discusses improvements in the boys' health.

Cystic Fibrosis (09:29)

In 2001, ten month old Kimberly experienced severe breathing problems; she was stabilized at the ER, and diagnosed with cystic fibrosis by a specialist; she has spent much time at the hospital. The genetic disease spurs the production of mucus, coating the lung's cilia, and leading to chronic infection; a membrane protein in airway cells normally acts as an ionic channel, but a DNA mutation blocks it. Therapy research produced Kalydeco, a faulty channel fixer; Kimberly was controversially treated, and rapidly improved.

Molly's Procedure (03:55)

After waiting eleven years to participate in a clinical trial, Molly's retinas were injected with a therapeutic virus; her vision is far better and disease progression has halted. She was treated before the disease caused irreversible damage; all trial patients improved.

Drug Approval and Ethics (05:28)

Austin and Max attend and speak at a Food and Drug Administration meeting, where patients and doctors advocate for approval of the Duchenne trial drug; the panel denies, but the final decision is not yet made. CRISPR precisely changes the sequence of DNA; there are three billion letters in the human genome, editing them is challenging. The technology could be used for trait selection and athletic advantage; doctors discuss the ethical issues of potential uses.

Timeline Researchers (01:55)

After Sonya was diagnosed with brain disease, she and her husband began researching; they quit their careers, got lab work, and were accepted to Harvard Medical. Their goal is to find a cure; they are tenacious, and progress in other gene therapy fields is encouraging.

Credits: The Gene Doctors (00:30)

Credits: The Gene Doctors

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The Gene Doctors

3-Year Streaming Price: $169.95



Every year, over a million babies are born worldwide with a hereditary disease—most are serious, and many are fatal. For much of history, doctors could only treat symptoms. Now an elite cadre of pioneers, gene doctors, is starting to target root causes. Through intimate stories of families whose lives are being transformed, The Gene Doctors takes viewers to the genesis of a medical revolution.

Length: 55 minutes

Item#: BVL166842

Copyright date: ©2017

Closed Captioned

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