Tag Archives: gene therapy

CRISPR: hitting the genetic bullseye

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Imagine throwing a trillion darts and having every single one hit the bullseye. Achieving this level of precision in gene editing would require highly intelligent delivery of the molecular machinery for DNA repair (CRISPR or one of its variants) into patients’ bodies, reaching only defective cells while bypassing healthy tissues. The benefits would be substantial: maximum therapeutic efficiency, zero waste, and reduced risks in terms of toxicity, immunogenicity, and unwanted mutations. How can such precise targeting be achieved? By acting on multiple levels, explain Jennifer Doudna and three researchers from her Innovative Genomics Institute. See their review article, Targeted delivery of genome editors in vivo in Nature Biotechnology.

Gene editing’s new bet on PKU

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Jennifer Doudna and Fyodor Urnov have founded a company that will take on the challenge of editing rare diseases, starting with phenylketonuria.

The problem is well known: many diseases are theoretically treatable by correcting the corresponding genetic defects with the help of CRISPR tools, but doing so risks being difficult or even impossible because of regulatory rigidity and economic unsustainability. The case of the first newborn treated with a bespoke therapy developed in record time (KJ Muldoon) was a splendid proof of principle. But it left many parents of children with rare diseases asking: when will it be our turn? At the same time, specialists have long been asking: will investors return to viewing gene editing as a profitable approach worth investing in?

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Beyond Baby KJ: manufacturing lessons for the next CRISPR cures

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The coordinated effort that last spring saved the life of little KJ Muldoon earned widespread and enthusiastic media coverage. But between the invention of the treatment and its delivery to the patient lay a lesser-told story: an unprecedented manufacturing sprint. Genetic Engineering & Biotechnology News organized an online roundtable led by its deputy editor in chief, Julianna LeMieux, to discuss how therapeutic components were produced quickly, cost-effectively, and to clinical-grade standards.

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CRISPR cures for kids? A new center is born!

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Jennifer Doudna and Priscilla Chan

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.

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Baby KJ is a symbol of hope — But is it replicable?

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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.

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Hi I’m JK, the first baby treated with a personalized CRISPR therapy

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The announcement was made yesterday at the annual meeting of the American Society of Gene & Cell Therapy and simultaneously published in the New England Journal of Medicine. I wrote about it for the Italian edition of Scientific American, but this story is worth seeing and reading.
PS: Before KJ was treated for his metabolic disorder, there was the case of Terry Horgan, who had muscular dystrophy. Unfortunately, the personalized treatment for him came too late, and he did not survive an adverse reaction. So yes, we can say that KJ represents a first: the first time genome editing was used early, rapidly, and tailored enough to truly offer hope for a happy ending.

CRISPR News – A Trio of Firsts

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Many interesting papers have been published recently; here are our top three picks. They cover an innovative gene therapy trial, a new experimental approach for oncology, and the development of novel tools to map gene enhancers.

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The Huge Little Thing: NanoCas is Coming

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3D structure of the NanoCas system [Mammoth Biosciences]

It is currently only a preprint on bioRxiv, but it has already attracted significant attention from the scientific community and the journal Science. Mammoth Biosciences, a company founded by CRISPR co-inventor Jennifer Doudna, has developed NanoCas, a mini-editor that is just one-third the size of traditional gene-editing scissors (Cas9).

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Chemotherapy pretreatment claims a victim in a CRISPR trial

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Busulfan 3D

Experimental patients often find themselves in a paradoxical situation: they must be sick enough to qualify for a clinical trial but healthy enough to endure its side effects. They also need the audacity to subject their bodies to protocols whose safety and efficacy remain unproven. For this reason, many describe them as pioneers or even warriors.

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CRISPR in Trump’s time

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A wait-and-see atmosphere lingers in the biotech and pharma sector. Vice President-designate J.D. Vance recently spoke with enthusiasm on Joe Rogan’s podcast about the first CRISPR treatment to hit the market—the one for sickle cell anemia. However, there’s rising concern about RFK Jr., who could exert significant influence over health and food policies. His broad “natural = good” ideology is both philosophically and scientifically dubious, and his clear anti-GMO positions are worrisome. Listening to his conversation with anti-biotech activist Jeffrey Smith on the RFK Jr. podcast reveals a revival of the classic 1990s narrative—corporations as villains, a corrupt establishment, suppressed scientists, concealed health risks—all now repurposed to criticize CRISPR technology, which is portrayed as unreliable and uncontrollable.