New Blood for Gene Therapy

A promising gene therapy trial, derailed by cancerous side effects in a young patient, is set to reboot with the help of next generation gene-transfer vectors.

| 4 min read

Register for free to listen to this article
Listen with Speechify
0:00
4:00
Share

Klein working with five-year old Felix Ott, who was diagnosed with Wiskott-Aldrich Syndrome at age three. When he was four, Felix received stem-cell gene therapy, and the now seven-year-old has since been able to live a normal life.VERENA MULLER

The two 3-year-olds were very, very sick. One was bleeding internally, suffered from severe eczema and anemia, and had multiple infections in his lungs and colon. The other had a dangerously low platelet count, recurring respiratory tract infections, and a life-threatening salmonella infection in his blood. Both turned up in pediatric hematologist Christoph Klein’s office in 2006, their parents clutching tightly to the hope that Klein might be able to save their children’s lives.

Klein, then at the Hannover Medical School in Germany, was running the first-ever gene therapy trial for Wiskott-Aldrich Syndrome, a rare and life-threatening disease caused by mutations in WAS, a gene whose protein product normally regulates the formation of actin polymers in hematopoietic cells. A dearth of functional WAS protein causes ...

Interested in reading more?

Become a Member of

The Scientist Logo
Receive full access to more than 35 years of archives, as well as TS Digest, digital editions of The Scientist, feature stories, and much more!
Already a member? Login Here

Keywords

Meet the Author

  • Megan Scudellari

    This person does not yet have a bio.
Share
Image of a woman in a microbiology lab whose hair is caught on fire from a Bunsen burner.
April 1, 2025, Issue 1

Bunsen Burners and Bad Hair Days

Lab safety rules dictate that one must tie back long hair. Rosemarie Hansen learned the hard way when an open flame turned her locks into a lesson.

View this Issue
Conceptual image of biochemical laboratory sample preparation showing glassware and chemical formulas in the foreground and a scientist holding a pipette in the background.

Taking the Guesswork Out of Quality Control Standards

sartorius logo
An illustration of PFAS bubbles in front of a blue sky with clouds.

PFAS: The Forever Chemicals

sartorius logo
Unlocking the Unattainable in Gene Construction

Unlocking the Unattainable in Gene Construction

dna-script-primarylogo-digital
Concept illustration of acoustic waves and ripples.

Comparing Analytical Solutions for High-Throughput Drug Discovery

sciex

Products

Atelerix

Atelerix signs exclusive agreement with MineBio to establish distribution channel for non-cryogenic cell preservation solutions in China

Green Cooling

Thermo Scientific™ Centrifuges with GreenCool Technology

Thermo Fisher Logo
Singleron Avatar

Singleron Biotechnologies and Hamilton Bonaduz AG Announce the Launch of Tensor to Advance Single Cell Sequencing Automation

Zymo Research Logo

Zymo Research Launches Research Grant to Empower Mapping the RNome