Tag Archives: Luigi Naldini

Chronicles from the London editing summit

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CRISPR patient Victoria Gray talking at the summit (credit The Royal Society)

The third – and perhaps final – act of the Human Genome Editing Initiative ended last week. The first summit (Washington 2015) was held amid enthusiasm for the invention of CRISPR, with the aim of fostering a constructive dialogue between science and society. The second edition (Hong Kong 2018) was dominated by the birth in China of the first edited human beings. The main points in the agenda of geneticists and bioethicists meeting a few days ago (London 2023) was to overcome the shock and focus on the next challenges: broadening the range of treatable diseases, reducing the costs of therapies, simplifying them so they can be administered anywhere in the world, and reach as many sick people as possible.

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Give gene therapy a market chance

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From Jakob Kamil Guziak’s website

The scientific renaissance is still there, but the commercial abandonnement is already going on. Patients affected by rare diseases and their families are worried they won’t be able to get treatments that are safe and effective but unprofitable for drugmakers. Take a look at the story of Jakob Kamil Guziak, recently told by Business Insider.

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Hit&Run – the Italian way to epi-editing

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The San Raffaele-Telethon Institute in Milan has been a leading player in gene therapy for many years. Nowadays, Angelo Lombardo, Luigi Naldini, and colleagues are making news with epigenetic editing. Their 2016 paper in Cell on hit-and-run epigenetic editing is considered seminal work. The company they co-founded, Chroma Medicine, received substantial financing, as recently reported by Nature Biotechnology. Last but not least, the presentation given in May at the annual meeting of the American Society of Gene and Cell Therapy inspired a Science news entitled “Better than CRISPR? Another way to fix gene problems may be safer and more versatile.” In brief, they injected mice to silence the expression of the PCSK9 gene, lowering “bad” cholesterol levels for months

About chromosomal mayhem in edited embryos

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Luigi Naldini, SR-Tiget

CRISPeR Frenzy asked Luigi Naldini of the San Raffaele Telethon Institute for Gene Therapy in Milan for comment on three studies published in June on the preprint server bioRxiv. The experiments were carried out independently by the groups of Kathy Niakan of the Francis Crick Institute in London, Dieter Egli of Columbia University in New York City, and Shoukhrat Mitalipov of Oregon Health & Science University in Portland. These findings heighten safety concerns about heritable genome editing (see the news item by Heidi Ledford in Nature). Below you can read Naldini’s thoughts.

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Do we need a Global Observatory for Gene Editing?

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osservatorio globale NatureEveryone knows IPCC, the forum created under the auspices of the United Nations to review the state of knowledge on climate change, draw scenarios on its impact, and compare alternative policies. Does the world need a similar body for the biotech revolution ahead, as claimed by Sheila Jasanoff and J. Benjamin Hurlbut in Nature? Is a Global Observatory on Gene Editing the solution to our CRISPR troubles? We asked a pioneer of gene therapy and a pioneer of gene drives, but also a bioethicist, a political scientist, a social psychologist, a science historian. Continue reading →

Gene therapy meets CRISPR

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gene therapyThe aim is engaging: to treat an increasing number of diseases by correcting the underlying genetic defects. And researchers are breathing optimism at last. The San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan has already treated 58 patients (including ADA-SCID, leukodystrophy, Wiskott-Aldrich syndrome and beta-thalassemia) and the count is approaching 300 worldwide. Moreover the promise of genome editing is looming on the horizon. We discussed the present and future of the field with the SR-Tiget director Luigi Naldini, who contributed to the latest report on human genome editing published by the US National Academies of Sciences and Medicine. Continue reading →