Combined Stem Cell-Gene Therapy: Shows Promise for In Vitro Genetic Cures

June 1, 2009

(ChattahBox)—The dizzying pace of stem cell research boggles the mind, as new and exciting medical discoveries offering potential cures for devastating genetic diseases are announced almost on a daily basis.

A groundbreaking study taking place at the Salk Institute for Biological Studies, in California, combining gene therapy to correct the defect with specialized pluripotent stem cells, may one day offer a cure for genetic diseases In Vitro, stopping the disease in its tracks.

There are more than 6000 known single-gene disorders, which occur in about 1 out of every 200 births. These statistics virtually guarantee that most of us would be personally affected in some way by a genetic disease. Single-gene disorders include diseases, such as Huntington disease, Sickle cell anemia, Cystic fibrosis, Duchenne muscular dystrophy, Spinal Muscular Atrophy and thousands more.

I specifically mention Spinal Muscular Atrophy, because my own grandchild was born with the devastating, often fatal motor neuron disease, commonly referred to as SMA Type 1. Most babies born with SMA Type 1 don’t live to see their second birthday. My grandson died three days before his first birthday from the heartbreaking effects of the disease.

My story and my grief are not unique. Consider the nearly 6000 genetic diseases that affect families from all walks of life across the globe, causing untold heartache, lifelong disability and early death.

The lead Salk Institute researchers for this exciting new stem cell treatment, Inder Verma and Juan Carlos Izpisúa Belmonte, pioneered the gene therapy and stem cell reprogramming techniques used in this promising study to cure genetic diseases.

If we can demonstrate that a combined iPS–gene therapy approach works in humans, then there is no limit to what we can do,” said Verma. The researchers cured a genetic disease in human cells, proving in principle for the first time, that their treatment works on humans.

“In theory we could transplant it into a human and cure the disease,” said Belmonte.

How did their treatment work? The researchers took hair or skin cells from patients with Fanconi anemia, a genetic blood borne disease, which causes bone marrow failure, leukemia and other cancers. The researchers then went to work to correct the genetic defect in the cells, using an advanced form of gene therapy pioneered by Dr. Verma.

Once the defect in the cell was corrected, the researchers then reprogrammed the cell into an induced pluripotent stem or iPS cell using a combination of transcription factors, OCT4, SOX2, KLF4 and cMYC.

IPS cells are an amazing scientific achievement, because they act like natural pluripotent stem cells, such as embryonic stem cells, which are vital in genetic cures, but without the need to use controversial embryonic cells.

The researchers then took the new iPS patient-specific iPS cells and transplanted them into a cell afflicted with Fanconi anemia. What happened next is the key to the success of future genetic cures. The IPS cells differentiated into hematopoietic progenitor cells, which targeted the unhealthy hematopoietic cells responsible for bone marrow failure.

Translation: the researchers cured the cell, wiping out the genes that caused bone marrow failure.

What’s next on the horizon for this successful groundbreaking study? Head researchers Belmonte and Verma need to conduct further studies to overcome the propensity of iPS cells to cause tumors, when certain viruses are used as a delivery system in gene therapy.

However, scientists have essentially overcome this hurdle by using an adenovirus as a delivery system. Belmonte and Verma used a unique delivery system by combining various protein based transcription factors.

This new treatment was made possible by a $6.6 million grant from the California Institute Regenerative Medicine, for research aimed at translating basic science into clinical cures.

After further studies and clinical trials, the new treatment could very well offer a potential cure for many genetic diseases in our lifetimes.

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  3. Motor-Neuron-Disease » MS Society Family & Friends Newsletter on June 9th, 2009 10:16 am

    […] Combined Stem Cell-Gene Therapy: Shows Promise for In Vitro Genetic … – Chattahbox.com(ChattahBox)—The dizzying pace of stem cell research boggles the mind, as new and exciting medical discoveries offering potential cures for devastating genetic diseases are announced almost on a daily basis. A groundbreaking study taking place at … […]

  4. Article Museum » Blog Archive » Combined Stem Cell-Gene Therapy: Shows Promise for In Vitro … on August 13th, 2009 5:48 pm

    […] the original post: Combined Stem Cell-Gene Therapy: Shows Promise for In Vitro …   0 Comments | Posted in Uncategorized, tagged: business, cell, mind, monitoring, […]

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