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CRISPR cures for kids? A new center is born!
Last May, the case of baby KJ made headlines: the child, suffering from a severe metabolic disorder, received a therapy developed specifically for him in just six months. The rapid improvement in his condition and his discharge from the hospital left the rare disease community with a pressing question: was this an unrepeatable one-off, or a replicable model of intervention? The right answer might be the latter, as demonstrated by the launch of the Center for Pediatric CRISPR Cures in California. This new center, to be led by Fyodor Urnov, begins with the mission of developing customized genome-editing treatments for eight young patients with congenital metabolic and immune system disorders.
Continue readingItalian Food for Thought
Food for Thought is a coalition of 18 Italian associations in the agrifood sector, established in 2017 to promote innovation in agriculture. Today, nearly eight years after the first manifesto, a new one has been presented to address the challenges of both the present and the future, including the climate crisis, geopolitical tensions, and changing consumption patterns.
The event was held yesterday in Rome at the initiative of Senator Bartolomeo Amidei, as part of the activities of the Parliamentary Intergroup on Made in Italy and Innovation.
Among the key points is the inclusion of New Genomic Techniques — a clear sign that there is widespread awareness within the Italian productive sector of the importance of genetic improvement, and that there are voices in the national Parliament willing to advocate for “innovative and sustainable agriculture.”
Gene drives: the first book by a pioneer
Italy has a strong tradition in malaria research, and it’s noteworthy that one of the most innovative tools to fight this disease in the future was developed with the crucial contribution of an Italian scientist. Andrea Crisanti has become a familiar face in the country due to his expertise in COVID-19 epidemiology and numerous TV appearances, first during the pandemic and later in his role as a senator. Yet his international reputation is more firmly rooted in a different field: the development of CRISPR-based strategies to eliminate malaria-transmitting mosquitoes.
Continue readingEvolving a new CRISPR system to insert genes
Recently, David Liu won the Breakthrough Prize for inventing two tools for precise, small-scale genome editing (base editing and prime editing). However, in some cases, rather than correcting a mutation within a defective gene, it may be more practical to insert a fully functional copy of the gene. That’s the mission of evoCAST, the latest invention from the Broad Institute near Boston, a hub for next-generation CRISPR tools.
Continue readingXenotransplants Edge Closer to Reality
There is still no consensus on the best way to humanize pig organs, but recent progress has convinced the Food and Drug Administration (FDA) to greenlight the first clinical trial.
The trial will begin enrolling six participants, and could expand to 44 if early results are promising. A six-month survival after the xenotransplant will be considered a success indicator, although it’s unclear how many patients will need to reach this milestone to win the agency’s approval.
Continue readingBaby KJ is a symbol of hope — But is it replicable?
The personalized editing therapy developed at record speed for the American newborn required preliminary studies, favorable circumstances, and a heroic collective effort.
After 307 days, little KJ Muldoon was discharged from the Children’s Hospital of Philadelphia (CHOP), wearing a tiny graduation outfit complete with a blue gown and cap. Born ten months ago with a severe metabolic disorder, the baby received a genome editing therapy developed exclusively for him, and his remarkable progress has been hailed by many as the dawn of a new era in precision medicine. A month after the publication of his case in the New England Journal of Medicine, we take a closer look at how researchers managed to develop the treatment in just six months—and whether this breakthrough could be replicated for other rare disease patients in need of life-saving therapies.
Continue readingWill edited plants be patentable in EU?
A year after the European Parliament voted to ban patents, EU countries still seek a compromise on NGT regulation
The revision of the regulatory framework for genetically modified plants currently underway in Europe aims to keep pace with technological advances and support the development of sustainable agriculture. The scientific community, the seed industry, and major farmers’ associations view the overall framework positively, but the devil is still in the details.
Continue readingFirst CRISPR treatments in Italy
Italy has begun administering the first CRISPR-based treatment. The therapy for sickle cell disease and beta thalassemia (Casgevy) has already been delivered to four patients across three clinical centers within a month. The announcement was made during the conference “Italian Primacy in the Treatment of Hemoglobinopathies” held yesterday at the Senate.
For more information, we recommend Francesca Ceradini’s article in Osservatorio Terapie Avanzate.
The Global Observatory for Genome Editing looks for a bioethics reset
A summit in Cambridge, Massachusetts, and a special issue of The CRISPR Journal featured a diversity of voices advocating for a broader debate in the name of cosmopolitanism and participation. How relevant is the call?
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