Blog | HemaCare

HemaCare Leukopaks Support Novel Gene Editing Strategy

May 7, 2019 10:07:00 AM / by Nancy Andon, MSc posted in leukopaks, gene editing, hematopoietic stem cells

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In an independent publication [1] researchers at UCLA cited using leukopaks obtained from HemaCare in order to investigate a new gene editing method.

The group is researching strategies to treat genetic blood disorders such as sickle cell anemia. While there has been a great deal of research focused on using CRISPR/Cas9 gene therapy to treat these illnesses [2], current strategies for gene editing in hematopoietic stem cells are inefficient, and thus costly.

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Improving the Efficiency of Clinical Gene Therapy

Jul 23, 2018 10:09:00 AM / by Nancy Andon, MSc posted in engraftment, gene therapy, gene editing, hematopoietic stem cells, progenitor cells

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An independent study [1] cites using HemaCare-sourced human cord blood, bone marrow, and mobilized peripheral blood as the starting material in a new strategy for more efficient genetic modification of human stem cells.

The study is based at the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Researchers there have been looking into ways to make gene transduction, which is used to introduce an edited copy of a human gene into stem cells, more efficient. The new strategy is part of their goal to improve the potency and long-term engraftment of stem cells used for gene therapy in the clinic.

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Scientists Grow Retinal Ganglion Cells from Stem Cells

Jan 27, 2016 1:00:04 PM / by Shweta posted in cellular reprogramming, CRISPR, gene editing, Innovation, research, stem cell transplantation, stem cells

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The gene editing tool CRISPR along with stem cells help scientists create retinal ganglion cells in the lab

Degeneration of retinal ganglion cells often leads to progressive and irreversible vision loss. These cells are the type of nerve cells located within the retina, which transmit visual signals from the eye to the brain. Retinal ganglion cells have limited intrinsic capacity to regenerate and cannot be replaced by new cells. Glaucoma and multiple sclerosis are the most common type of optic neuropathies that lead to vision loss and blindness due to death of retinal ganglion cells.

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Repairing the Genetic Mutation Caused by Duchenne Muscular Dystrophy

Feb 18, 2015 1:00:55 PM / by Shweta posted in gene therapy, genetic defects, gene editing, induced pluripotent stem cells, iPSCs, research, stem cells

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Scientists corrected the defective genes of Duchenne muscular dystrophy disease by genome editing

Duchenne muscular dystrophy (DMD) is a devastating progressive disease that usually starts in early childhood. DMD occurs because of a mutated gene which fails to produce dystrophin, a protein which is important for the normal structure and function of muscle. Currently, no definitive treatments are available except for palliative therapy, which can only delay the symptoms of the disease. Moreover, available treatments are not completely effective, as they treat just one aspect of the disease, and they may have side effects in the long run.

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