Pluripotent cells treat anemia?

Skin cells reprogrammed for pluripotency can be used to treat anemia in a mouse model of the disease, reports a study published online in Science today (December 6). The researchers, led by linkurl:Rudolph Jaenisch;http://www.wi.mit.edu/research/faculty/jaenisch.html and Jacob Hanna at MIT, say the study provides proof of principle that linkurl:induced pluripotent stem cells;http://www.the-scientist.com/blog/display/53873/ (iPS cells) can be used to treat diseases. The scientists first creat

| 1 min read

Register for free to listen to this article
Listen with Speechify
0:00
1:00
Share
Skin cells reprogrammed for pluripotency can be used to treat anemia in a mouse model of the disease, reports a study published online in Science today (December 6). The researchers, led by linkurl:Rudolph Jaenisch;http://www.wi.mit.edu/research/faculty/jaenisch.html and Jacob Hanna at MIT, say the study provides proof of principle that linkurl:induced pluripotent stem cells;http://www.the-scientist.com/blog/display/53873/ (iPS cells) can be used to treat diseases. The scientists first created "humanized" mouse models for anemia by using gene knock-in techniques to replace mouse hemoglobin genes with human versions that cause the disease. Mice homozygous for the allele developed anemia symptoms and died within 18 months. They used four transcription factors -- Oct4, Sox2, Klf4 and c-Myc -- to generate lines of iPS cells from skin cells of the anemic mice. They then "corrected" these cells by replacing the diseased gene with its wild-type counterpart using homologous recombination techniques -- an approach that has been linkurl:used;http://www.ncbi.nlm.nih.gov/sites/entrez?orig_db=PubMed&db=PubMed&cmd=Search&term=Blood%5BJour%5D%20AND%20108%5Bvolume%5D%20AND%201183%5Bpage%5D%20AND%202006%5Bpdat%5D%20AND%20Wu%5Bauthor%5D with ES cells. According to the paper, it worked comparably well in iPS cells. In the final step, they transplanted blood cells differentiated from the corrected iPS cells into three anemic mice. Using PCR, they showed that the DNA of the treated mice carried both the diseased and the corrected genes; a range of blood tests demonstrated that disease symptoms had improved to a state "comparable to control mice." But the researchers warn that major issues with the technique remain to be addressed before it can be used in treatment. One of the transcription factors they used in reprogramming iPS cells is believed to linkurl:cause tumors,;http://www.the-scientist.com/blog/display/53946/ and using retroviral vectors may also cause cancer.
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

Meet the Author

  • Alla Katsnelson

    This person does not yet have a bio.
Share
3D illustration of a gold lipid nanoparticle with pink nucleic acid inside of it. Purple and teal spikes stick out from the lipid bilayer representing polyethylene glycol.
February 2025, Issue 1

A Nanoparticle Delivery System for Gene Therapy

A reimagined lipid vehicle for nucleic acids could overcome the limitations of current vectors.

View this Issue
Enhancing Therapeutic Antibody Discovery with Cross-Platform Workflows

Enhancing Therapeutic Antibody Discovery with Cross-Platform Workflows

sartorius logo
Considerations for Cell-Based Assays in Immuno-Oncology Research

Considerations for Cell-Based Assays in Immuno-Oncology Research

Lonza
An illustration of animal and tree silhouettes.

From Water Bears to Grizzly Bears: Unusual Animal Models

Taconic Biosciences
Sex Differences in Neurological Research

Sex Differences in Neurological Research

bit.bio logo

Products

Photo of a researcher overseeing large scale production processes in a laboratory.

Scaling Lentiviral Vector Manufacturing for Optimal Productivity

Thermo Fisher Logo
Collage-style urban graphic of wastewater surveillance and treatment

Putting Pathogens to the Test with Wastewater Surveillance

An illustration of an mRNA molecule in front of a multicolored background.

Generating High-Quality mRNA for In Vivo Delivery with lipid nanoparticles

Thermo Fisher Logo
Tecan Logo

Tecan introduces Veya: bringing digital, scalable automation to labs worldwide