Scientists studying how genetics impact brain disease have long sought a better experimental model. Cultures of genetically-modified cell lines can reveal some clues to how certain genes influence the development of psychiatric disorders and brain cancers. But such models cannot offer the true-to-form look at brain function that can be provided by genetically-modified mice.
Even then, carefully breeding mice to study how genes impact the brain has several drawbacks. The breeding cycles are lengthy and costly, and the desired gene specificity can only be verified — but not guaranteed — when mouse pups are born.
In today’s Nature, scientists from Boston Children’s Hospital and UC San Francisco describe a new way to create customized mouse models for studying the brain. …
As the African-American civil rights movement was flowering in the 1960s, a less visible civil rights movement was dawning. And so was a revolution in science that may outshine that spurred by the U.S. space program.
It was a time when children with what is now called intellectual disability (ID) or developmental disability (DD) were “excused” from school and routinely abandoned to institutions. “Schools” like the Fernald Center in Massachusetts and the Willowbrook State School in New York housed thousands of residents.
In 1962, President John F. Kennedy convened a panel to propose a “National Action to Combat Mental Retardation,” at the strong urging of his sister Eunice Kennedy Shriver. Three weeks before JFK’s assassination, the first legislation passed. It changed the course of history. …
Many migrant children separated from their parents at the U.S. border, some of them very young, have landed in shelters where they often experience stress, neglect and minimal social and cognitive stimulation. The latest findings of the long-running Bucharest Early Intervention Project (BEIP), involving children in Romanian orphanages, tells a cautionary tale about the psychiatric and social risks of long-term deprivation and separation from parents.
BEIP has shown that children reared in very stark institutional settings, with severe social deprivation and neglect, are at risk for cognitive problems, depression, anxiety, disruptive behavior and attention-deficit hyperactivity disorder. But BEIP has also shown that placing children with quality foster families can mitigate some of these effects, if it’s done early.
The new BEIP study, published this week by JAMA Psychiatry, asked what happens to the mental health of institutionalized children as they transition to adolescence. Outcomes at ages 8, 12 and 16 suggest diverging trajectories between children who remained in institutions versus those randomly chosen for placement with carefully vetted foster families. …
Scientists around the world have been trying to replace damaged heart tissue using lab-made heart-muscle cells, either injecting them into the heart or applying patches laced with the cells. But results to date have been underwhelming.
“If you make cardiomyocytes in a dish from pluripotent stem cells, they will engraft in the heart and form muscle,” says William Pu, MD, director of Basic and Translational Cardiovascular Research at Boston Children’s Hospital. “But the muscle doesn’t work very well because the myocytes are stuck in an immature stage.” …
How sensitive are hospitals to the needs of lesbian, gay, bisexual and transgender (LGBT) patients? In a 2010 survey by Lambda Legal, 70 percent of transgender patients and 56 percent of gay/lesbian/bisexual patients reported discrimination from health care providers. Clinicians refused to provide needed care, refused to touch them or used excessive precautions, blamed them for their health status, were verbally abusive or were physically violent.
“Information from social media and other online sources can help us gain authentic and unsolicited accounts from vulnerable patient groups, like LGBT individuals who are not typically represented,” says Hswen.
Based on the tweets, the team determined which hospitals were more supportive of LGBT patients (the blue dots in the above map) and which were less supportive (the red dots).
The identified tweets included Twitter handles from 653 hospitals and contained LGBT-related terms: LGBT, transgender, trans, intersex, sex change, transisbeautiful, tranny, drag queen, preferred pronoun, transhealth, genderodyssey, cis, gay, lesbian, queer, rainbowhealth, gender fluid, homosexual, bisexual, homo, homophob and transphobe. A tweet classed as supportive might read, “@Hospital is hosting a LGBT resource fair;” a negative tweet might read: “Having sex with men does not mean I deserve less @Hospital.”
For 30 years, researchers have tried to develop an HIV vaccine that would stop the virus from gaining a foothold in the body — before it attaches to T cells and slowly weakens the immune system.
“It has been extremely challenging to induce effective antibody responses against HIV-1,” says Bing Chen, PhD, who researches HIV’s molecular mechanisms at Boston Children’s Hospital.
HIV offers just one target for a vaccine to mimic to trigger protective antibodies: the envelope protein on its surface. Scientists have been struggling to capture the complex protein’s precise structure — and specifically, its structure before the virus fuses with the T-cell membrane. …
The lab of Leonard Zon, MD, has long been interested in making blood stem cells in quantity for therapeutic purposes. To test for their presence in zebrafish, their go-to research model, they turned to the MYB gene, a marker of blood stem cells. To spot the cells, Joseph Mandelbaum, a PhD candidate in the lab, attached a fluorescent green tag to MYB that made it easily visible in transparent zebrafish embryos.
“It was a real workhorse line for us,” says Zon, who directs the Stem Cell Research Program at Boston Children’s Hospital.
In addition to being a marker of blood stem cells, MYB is an oncogene. About five years ago, Zon was having lunch at a cancer meeting and, serendipitously, sat next to Jeff Kaufman, who was also interested in MYB. Kaufman was excited to hear about Zon’s fluorescing MYB zebrafish, which can be studied at scale and are surprisingly similar to humans genetically.
“Have you ever heard of adenoid cystic carcinoma?” he asked Zon. …
Most of the time, cancer cells do a combination of two things: they overexpress genes that drive tumor growth and they lose normal genes that typically suppress tumors. No two tumors are exactly alike, but some combination of these two effects is usually what results in cancer. Now, for the first time, researchers have shown that it’s possible to treat cancer by delivering a gene that naturally suppresses tumors.
Researchers from Boston Children’s Hospital, Brigham and Women’s Hospital and Memorial Sloan Kettering Cancer Center combined their cancer biology and nanomaterials expertise and developed a therapeutic capable of delivering a tumor suppressor gene known as PTEN, the loss of which can allow tumors to grow unchecked.
In several preclinical models, their PTEN–boosting therapeutic was able to inhibit tumor growth. Their findings were published yesterday in Nature Biomedical Engineering. …