Amanda Mauro, PhD

The plan to make more custom CRISPR therapies — with Kiran Musunuru and Rebecca Ahrens-Nicklas In February, a team of scientists achieved a long-held scientific goal — creating a bespoke CRISPR editing therapy to treat a single patient with a unique genetic mutation. “Baby KJ” got his own custo...

🎙️Rebecca Ahrens-Nicklas, a pediatrician and gene therapy scientist at CHOP, and Kiran Musunuru, a UPenn gene editing scientist, join Post-Hoc Live to talk about the next steps to replicate "Baby KJ"'s bespoke CRISPR editing therapy. Watch live at 9.45am ET.
www.youtube.com/live/ig4gEOa...

Image that says "The Cambridge Commission on Immigrant Rights and Citizenship (CIRC) and the Massachusetts Office of Refugees and Immigrants present two free virtual workshops with practical tools for bystanders to intervene and de-escalate situations. Learn more about: ICE enforcement basics Community support roles What to do if detention occurs Please register for one of the following sessions for a Zoom link: - Wednesday, October 29, 6-7 p.m. This workshop will be conducted only in English - Wednesday, November 5, 6-7 p.m. Interpretation upon request. To request an interpreter, please use the registration link or email accesshelp@cambridgema.gov by Monday, October 27.""

The Cambridge Commission on Immigrant Rights and Citizenship and the MA Office of Refugees and Immigrants present 2 virtual workshops with practical tools for bystanders to intervene and de-escalate situations.

@theharvardcrimson.bsky.social @cambridgeday.com

www.cambridgema.gov/Departments/...

Mass. resources for those losing SNAP benefits during shutdown With more than 1 million Massachusetts residents expected to lose access to their SNAP benefits next month, these local resources may be able to offer support.

Mass. resources for those losing SNAP benefits during shutdown | Click on the image to read the full story

Exclusive: Rebooting dead human brains, a biotech startup seeks to reinvent early drug testing work Startup Bexorg, led by Zvonimir Vrselja, keeps human brains alive to test drugs. Their BrainEx system sustains brain cells for 24 hours to improve drug development and reduce animal testing.

Earlier this year I visited Bexorg to see its brain-in-a-bucket experiments in person. The approach raises many technical and ethical questions which I dug into in my profile of the startup in June. Read more here:
endpoints.news/rebooting-de...

Yale spinout raises $23M to test drugs on ‘rebooted’ human brains from organ donors Bexorg has collected $23 million in a Series A raise to scale up its human brain experiments, bringing its total funding to $42.5 million, the startup announced Wednesday.

The Yale spinout Bexorg has raised a $23M series A for its cutting-edge and unconventional approch to preclinical research: testing drugs on ‘rebooted’ human brains from organ donors. My story for @endpts.com has the details: endpoints.news/bexorg-raise...

Everett 13-year-old arrested by ICE and sent to Virginia detention facility By Marcela Rodrigues Globe Staff,Updated October 12, 2025, 44 minutes ago 31 A 13-year-old boy was arrested by ICE in Everett and sent to a juvenile detention facility in Virginia. A 13-year-old boy was arrested by ICE in Everett and sent to a juvenile detention facility in Virginia. A 13-year-old boy was arrested by Immigration and Customs Enforcement agents in Everett after an interaction with members of the Everett Police Department and sent to a juvenile detention facility in Virginia, according to his mother and immigration lawyer Andrew Lattarulo. The boy’s mother, Josiele Berto, was called to pick her son up from the Everett Police Department on Thursday, the day he was arrested. After waiting for about an hour and a half, she was told her son was taken by ICE, Berto told the Globe in a phone interview. “My world collapsed,” Berto said in Portuguese. From the police department, the boy was taken to ICE’s holding facility in Burlington on Thursday evening, where he spent a night before being transferred by car to the Northwestern Regional Juvenile Detention Center in Winchester, Va., on Friday morning, his mother said. The juvenile facility is more than 500 miles away from Everett. The boy is a 7th-grader at Albert N. Parlin School in Everett, his mother said. The teen and his family, who are Brazilian nationals, have a pending asylum case and are authorized to work legally in the United States, Lattarulo said.

ICE kidnapped a 7th-grader with a pending asylum claim and spirited him out of state without notifying his parents, seemingly with the cooperation of the local police in Everett, MA.

www.bostonglobe.com/2025/10/12/m...

Andrew Cuomo tweet: @ZohranKMamdani's "plan" provides no relief for working families who take the subway, and unnecessary benefits to wealthy New Yorkers who take the bus and can afford the fare. I focus on working families, while @ZohranKMamdani the mansion boy focuses on helping the rich.

[Me, a poor, on the subway, glowering jealously at all those fat cat, fancy pants rich people on the public bus]

Nexstar is trying to close a multi-billion dollar merger with Tegna. It requires the approval of Trump's FCC.

A Black man and a homeless man were found hanging from trees in Mississippi yesterday, for those who haven’t seen yet because it’s barely made it out of local news.

Potential threats trigger lockdowns at several Historically Black Colleges and Universities across the South Originally Published: 11 SEP 25 14:36 ET By Alisha Ebrahimji, CNN (CNN) — Multiple Historically Black Universities and Colleges in the South were on lockdown or have cancelled classes on Thursday…

Hampton University, Virginia State University, Bethune Cookman University (BCU) and Alabama State University were all on lockdown after receiving “potential threats to campus safety,” according to posts on school social media pages. buff.ly/yK7CDN1

NYC Mass Shooting Was Nearly Impossible to Prevent, Experts Say

Holy shit, they did it. They wrote the headline.

Brain endothelial gap junction coupling enables rapid vasodilation propagation during neurovascular coupling Vasodilatory signals are rapidly communicated across long distances by endothelial-endothelial gap junctions, enabling coordinated dilation of the arterial network during neurovascular coupling.

Very cool new paper by @thegulab.bsky.social published in Cell, showing that arterial endothelial coupling via gap junctions determines the speed and spread of vasodilation induced by neuronal activity ! #cerebrovascular #bloodflow #endothelium

Massive proteomics study reveals new clues about Alzheimer’s and rekindles old ones Amyloid plaques and tau tangles are the most well-known protein structures associated with Alzheimer’s, but a new study bolsters the longstanding notion that there’s a lot more going on in the disease...

A massive proteomics study analyzed 250 million protein markers from 18,600 people, uncovering new — and familiar — clues about Alzheimer’s and other neurodegenerative diseases.
endpoints.news/massive-prot...

Exclusive: FDA's Makary sought rejection of KalVista’s drug in an unusual move by commissioner, sources say FDA Commissioner Marty Makary reportedly attempted to reject KalVista Pharmaceuticals' drug application, sources said. An HHS spokesperson calls the claim 'totally false.'

Exclusive: FDA's Makary sought rejection of KalVista’s drug in an unusual move by commissioner, sources say
endpoints.news/sources-fda-...

The effect of transient sex hormone fluctuations on endothelial function | American Journal of Physiology-Heart and Circulatory Physiology | American Physiological Society This review article summarizes the current literature investigating the effects of transient sex hormone fluctuations on large artery endothelial function, primarily concerning the menstrual cycle and the diurnal rhythm of testosterone secretion. Women and men experience acute fluctuations in circulating levels of sex hormones, and there is substantial variability in circulating levels of sex hormones in both sexes. These acute fluctuations in sex hormones generally coincide with alterations in endothelial cell function and in vivo endothelium-dependent vasodilation, and we see that the timing of these acute fluctuations match the timing of cardiovascular events in both women and men. It is important to improve our understanding of how acute fluctuations in sex hormones affect endothelial function in women and men, as clinical cardiovascular complications coincide with these changes. This would allow for the identification of novel therapeutic targets and aid in the prevention and treatment of cardiovascular disease.

The second reviews the literature assessing the effect of acute fluctuations in sex hormones on large artery endothelial function, including across the menstrual cycle and the diurnal cycle of testosterone secretion journals.physiology.org/doi/abs/10.1... 3/3

Mineralocorticoid and estrogen receptors as sex-dependent modulators of vascular health in aging and obesity Cardiovascular disease (CVD) is the leading cause of death in both men and women, but there are sex differences in the timing and mechanisms of diseas…

The first reviews the literature assessing sex-specific mechanisms of how the mineralocorticoid and estrogen receptors contribute to vascular outcomes in aging and obesity www.sciencedirect.com/science/arti... 2/3

A scene from Always Sunny where Mac hands Dennis a plate of something, and Dennis tosses it down the hallway. Mac is labeled "literally every company" the plate is labeled "AI" and Dennis is labeled "Me"

I don't think any single piece of information has ever radicalised me like reading the statistics on how many women have their husbands/boyfriends dump them because they got a terminal illness diagnosis

Cocaine vaccine, a longshot attempt to quell cravings, yields mixed data in small study Weill Cornell Medicine study shows cocaine vaccine triggers antibodies & reduces drug use in small trial, but effects modest. Monthly shots helped 21 addicts, says researcher Ronald Crystal.

Here's a wild idea: attach a cocaine-like molecule to the surface of a torn-up adenovirus and inject it in chronic drug users to help them develop antibodies that prevent cocaine from reaching the brain.

I have the details of the first clinical results in @endpts.com

endpts.com/researchers-...

CRISPR Therapeutics partners with siRNA company, showing that gene editing isn’t the only answer The gene editing company CRISPR Therapeutics surprisingly struck a deal with China-based biotech Sirius Therapeutics to jointly develop its experimental gene-silencing siRNA drug for blood clotting di...

Crispr Therapeutics surprised me yesterday when it partnered with an siRNA company. After all, isn't CRISPR supposed to do anything that siRNA can do, only permanently? I talked to CEO Sam Kulkarni about why even he doesn't think gene editing is a panacea. @endpts.com

endpts.com/crispr-thera...

David Liu, Sam Sternberg unveil new way to insert big genes in human cells Scientists led by David Liu and Samuel Sternberg develop improved CRISPR-based gene insertion tool called evoCAST, boosting editing efficiency in human cells to 10-40%.

In a much anticipated update for the gene insertion field, David Liu and Samuel Sternberg have figured out how to get #CRISPR associated transposases (CASTs) to insert full genes efficiently in human cells. @leileiwu.bsky.social and I have the story for @endpts.com

endpts.com/david-liu-sa...

The inside story of the six-month sprint to create a custom CRISPR therapy to fix one infant’s rare disease Two researchers have been working to rapidly design custom CRISPR treatments for rare diseases that profit-driven drug companies have left behind. But one child's dire condition provided an opportunit...

BREAKING NEWS: Last August, scientists began creating a custom #CRISPR therapy to fix a mutation responsible for one baby's debilitating disease. He was treated in February and doing better. My deep dive for @endpts.com had the details: endpts.com/philadelphia...

Trump officials take steps toward a radically different NSF Efforts to shrink staff, budget, and focus have alarmed members of Congress

Another scoop from Jeff Mervis (@policyhound.bsky.social): NSF's ~1400 grant terminations have disproportionately affected PIs from groups underrepresented in science: women, racial & ethnic minorities, & those with disabilities. 1/3
www.science.org/content/arti...

'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis Fyodor Urnov and Kiran Musunuru advocate for streamlined CRISPR treatments for rare genetic diseases, partnering with Danaher and CHOP to develop custom therapies for HLH and metabolic disorders.

#CRISPR isn't living up to its potential. Thousands of rare diseases that could be treated with gene editing remain untouched by drug companies. Two scientists, @urnov.bsky.social and @kiranmusunuru.bsky.social, have a plan to fix that.

Read more in my story @endpts.com: endpts.com/crispr-isnt-...

CRISPR Therapeutics' gene editing therapy reduces bad cholesterol and triglycerides by as much as 80% CRISPR Therapeutics has revealed promising but early data from its first clinical study of a gene editing therapy to lower the risk of heart disease, setting the stage for competition ...

The first results are in from CRISPR Therapeutics' gene editing therapy targeting ANGPTL3 to prevent heart disease. The treatment reduced LDL cholesterol and triglycerides by as much as 80%. Read more in my story for @endpts.com

endpts.com/crispr-thera...

Exclusive: Vertex drops AAV gene therapy research, tRNA therapy partnership Vertex is pulling back on its genetic medicine research, ending all work related to adeno-associated viruses.

Vertex has dropped its #AAV gene therapy research, impacting partnerships with Affinia Therapeutics and tRNA therapy startup Tevard Biosciences, and raising questions about its #CRISPR therapies for muscular dystrophy. Read more in my scoop for @endpts.com endpts.com/vertex-drops...

Exclusive: Vertex drops AAV gene therapy research, tRNA therapy partnership Vertex is pulling back on its genetic medicine research, ending all work related to adeno-associated viruses.

Exclusive: Vertex is pulling back on its genetic medicine research, ending all work related to adeno-associated viruses, or AAVs

The inside story of how SalioGen, a startup trying to wrangle ‘jumping genes’ to make medicines, fell apart SalioGen Therapeutics, led by Ray Tabibiazar, shut down after failing to perfect its bat-derived gene editing technology. Despite raising $115M, the company struggled with targeting and delivery issue...

Teslas. Vacations. “Jumping genes.” SalioGen dangled big perks to crack its gene insertion tech. It shut down instead. @scienceboss.bsky.social digs into how the startup fell apart.
endpts.com/how-saliogen...