Tag Archives: gene therapy

WHO’s roadmap on genome editing

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A multi-disciplinary panel of 18 experts from all over the world, a two years long consultation, over 150 pages. The much-awaited report of the World Health Organization on human genome editing was delivered on July 12 and is divided into three parts: A framework for governance, Recommendations, and Position Paper. While not legally binding, it is expected to influence both governments and the scientific community, by offering a roadmap based on widely shared ethical principles and usable policy tools.

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Toward an NIH-validated CRISPR toolkit

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The Somatic Cell Genome Editing (SCGE) Consortium is working to accelerate the development of better methods of editing. Seventy-two principal investigators from 38 institutions are pursuing 45 distinct but well-integrated projects, funded by the US National Institutes of Health with US$190 million over 6 years. A perspective published in Nature details their plans:

“New genome editors, delivery technologies and methods for tracking edited cells in vivo, as well as newly developed animal models and human biological systems, will be assembled—along with validated datasets—into an SCGE Toolkit, which will be disseminated widely to the biomedical research community. We visualize this toolkit—and the knowledge generated by its applications—as a means to accelerate the clinical development of new therapies for a wide range of conditions”.

Great progeria paper opens CRISPR new year

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A paper published in Nature by CRISPR innovator David Liu and a giant in medical genetics, Francis Collins, raises great hopes for treating a rare, devastating pediatric disease causing premature-aging (Hutchinson-Gilford progeria syndrome). “The outcome is incredible,” according to gene-therapy researcher Guangping Gao. “Dance on the lab bench” amazing, according to editing pioneer Fyodor Urnov. Let’s be clear: the CRISPR variant called a base-editor has helped only progeria mice so far, but results are beyond anyone’s wildest expectations. One injection is enough to fix the single-letter mutation in several tissues, doubling mice’s lifespan. To learn more, see David Liu’s tweets and the NIH Director’s Blog.

Our CRISPR future, according to J. Doudna

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The Nobel Prize for CRISPR is one of the most exciting ever assigned in chemistry and one of the most celebrated in the media, for reasons related to the invention and the inventors alike. On the one hand, the technique is changing the practice and the image of genetic engineering. On the other hand, Jennifer Doudna and Emmanuelle Charpentier are not merely great scientists; they are a success story in cracking the glass ceiling and a symbol of the strength of collaboration.

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Breaking or fixing? A tale of two approaches for hemoglobinopathies

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Painting by Hertz Nazaire

Covid19 is affecting everyone, but it has hit the sickle cell (SCD) community particularly hard. According to STAT News the pandemic has temporarily stopped clinical trials and the introduction of new drugs, besides directly impacting SCD patients who are at high risk for severe complications from Sars-Cov2 infection and may need hospital assistance for SCD pain crises.

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Modulation better than correction. A new CRISPR paradigm is emerging

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ronald-cohn3
Ronald Cohn (SickKids)

Another CRISPR step in the way out of congenital muscular dystrophy type 1A (MDC1A) is announced by Ronald Cohn and colleagues in Nature this week. This is still preclinical research in mice, but the indirect approach presented by the Canadian team holds great promise.

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CRISPR breathes new life into fetal lungs

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Researchers from Penn Medicine and Children’s Hospital of Philadelphia have fixed a lethal mutation in the prenatal mouse models of a rare pulmonary disease. The hope is that the approach of in utero editing described in Science Translational Medicine will work for other congenital lung diseases as well.

I asked one of the corresponding authors, Edward Morrison, scientific director of the Penn’s Institute for Regenerative Medicine, to explain what they have done and what to expect next. See Q&A below. Continue reading →

Blood stem cells getting ready for editing

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editing genomicoDo you remember the alarm about the cancer risks of CRISPR? According to a study published in Cell Stem Cell by Italian researchers, the DNA-damage response is not such a big problem in edited cells. At least not in hematopoietic stem cells edited with highly specific nucleases. Continue reading →

Editing Duchenne. Where are we now?

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Duchenne CRISPR

© STEVE GRAEPEL (“Infographic: Treating Duchenne Muscular Dystrophy with CRISPR“, The Scientist, September 2018)

In 2013, multiple labs sharpened CRISPR molecular scissors against Duchenne muscular dystrophy, using cells from patients in vitro. In 2016, the dystrophin gene was successfully edited in mice. Then last summer, Eric Olson did it in dogs . Where are we now in the struggle to cure this severe type of myopathy afflicting 300,000 boys in the world? What is still needed to move into a clinical trial? Continue reading →

CRISPRing in the womb

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pregnant-2640994_960_720It’s another CRISPR first: fixing a hereditary disease in utero. Scientists from Pennsylvania University and the Children’s Hospital of Philadelphia used a base editor to rescue tyrosinemia in fetal mice. “The results of this proof-of-concept work demonstrate the possibility of efficiently performing gene editing before birth, pointing to a potential new therapeutic approach for selected congenital disorders,” Avery C. Rossidis and colleagues write in Nature Medicine.  Continue reading →