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Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease

ROCKVILLE, Md., Oct. 13, 2020 /PRNewswire/ — Foundation to Fight H-ABC, a non-profit organization dedicated to increasing awareness and driving development of a cure for the degenerative children’s disease, H-ABC, today announced a sponsored research agreement with the University of Massachusetts Medical School and Yale University to advance a targeted gene therapy for H-ABC.

“We have high hopes to quickly prove efficacy with this approach to move research forward and find a permanent cure for this devastating disease,” said Michele Sloan, Co-Founder, Foundation to Fight H-ABC.

H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum) belongs to a group of conditions called leukodystrophies, diseases that affect the white matter of the brain. These diseases disrupt the growth or maintenance of the myelin sheath, a protective layer that insulates nerve cells and allows for the transmission of messages between cells.

Caused by a mutation in the TUBB4A gene, H-ABC is a rare genetic disorder that affects certain parts of the brain—specifically the basal ganglia and the cerebellum, which control movement. H-ABC targets these important structures, reducing both their size and function. As a result, children who suffer from H-ABC often experience motor problems, cannot walk, talk, or sit on their own. Currently, there is no known cure for this disabling and life-threatening condition.

The teams of Dr. Guangping Gao (University of Massachusetts Medical School) and Dr. Karel Liem (Yale School of Medicine) will combine extensive expertise in the fields of Adeno-associated virus (AAV), a platform for gene delivery for the treatment of a variety of human diseases and H-ABC disease models, to develop AAV vectors to silence or outcompete the mutated TUBB4A gene.

“To date, AAV-based gene delivery system is the vector of choice for in vivo gene therapy of many currently untreatable rare diseases including H-ABC,” said Guangping Gao, Ph.D. “We are very excited for starting close collaborations with Dr. Liem’s team at Yale and the Foundation to Fight H-ABC to develop potential gene therapeutics for this devastating disease.”

“With the support from the Foundation to Fight H-ABC, we are excited to build upon our mechanistic studies of the disease and to collaborate with Dr. Gao of the University of Massachusetts to develop and test AAV approaches to H-ABC,” said Karel F Liem Jr., M.D., Ph.D.

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Joseph M. Cronin, first Massachusetts secretary of education, dies at 85

“In order to really give poor people in the inner city a chance to compete,” he told the Globe, “we will have to spend more on their education than on the average child in other communities.”

Dr. Cronin, who in his long, multifaceted career as an educator had also served as president of what is now Bentley University, died Saturday in the Pat Roche Hospice Home in Hingham of progressive supranuclear palsy. He was 85 and had lived in Milton for many years.

As he prepared to retire in 1997 from leading what was then Bentley College, he received a letter from nearly 20 colleagues who signed themselves as “the faculty and staff of color.”

“Under your leadership diversity has become a business imperative for the college,” they wrote. “Your leadership in diversity has resulted in many of us joining the Bentley community.”

When Dr. Cronin first arrived in 1991 to serve as president, he stressed that he wanted the college to prepare graduates to be global thinkers ready for careers anywhere in the world.

“Businesses want people who are versatile, who can go, say, to Zimbabwe for a week on a special assignment, and they had better have had courses in government and history to absorb this,” Dr. Cronin told the Globe. “We want them to be ready.”

As Massachusetts secretary of education in the early 1970s, he played a key role in implementing Chapter 766, the state’s special education law that became a model for legislation in other states.

Dr. Cronin also was credited with increasing state support of the arts and humanities in public education, from $250,000 to $2.5 million

He served as state secretary of education for three years before leaving to become superintendent of schools in Illinois. As with the education secretary post, he was the first to fill that newly created position.

In Illinois, he was hired by and answered to a recently formed state Board of Education and he stayed until 1980.

“The biggest dream the board had was to desegregate the remaining 30 city school districts. By 1980, 20 of them did so,” Dr. Cronin wrote in 1981.

The efforts by Dr. Cronin and the state board “won us mixed reviews,” he noted in the 25th anniversary report of his Harvard class.

The Chicago Reader, an alternative weekly, “said we kicked the door of racial segregation down” in persuading Chicago’s school officials to desegregation numerous schools.

But the Chicago Tribune, he added, “called me ‘one of the most imperious and extravagant bureaucrats in the history of Illinois,’ which prose I found somewhat extravagant.”

Joseph Marr Cronin was born in Dorchester on Aug. 30, 1935, the oldest of four siblings.

His father, Joseph Michael Cronin, was an accountant and an attorney. His mother, Mary Marr Cronin, had been a secretary for the Marr family’s construction business.

Dr. Cronin’s family moved to Milton when he was a boy and he graduated from Boston College High School in 1952.

He went to Harvard College, where

Total of 106 students, 57 staffers test positive for COVID in Massachusetts schools over the last week, education officials report

Massachusetts school districts have reported 106 new coronavirus cases over the last week among students who are learning in-person or through hybrid instruction, according to the Massachusetts Department of Elementary and Secondary Education.


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Additionally, DESE reports 57 new COVID-19 cases among district staff members. The new cases reflect reporting between Oct. 1 through Oct. 7 across school districts, charter schools, collaboratives and approved special education schools.

The data includes positive cases for students in hybrid or in-person learning models, excluding students in districts that are learning only remotely. Staff cases include employees who have been in a district building within the seven days before the report of the positive case.

Notably, there were eight new cases among students in Haverhill schools, five among students in Hudson schools and Burlington schools and four among students in Hingham schools. Every other district saw three or fewer new cases, with the vast majority reporting none.

There were six reported cases of the virus among staff members in Milton schools, with two COVID-positive students as well.

Tyngsborough schools report three staff members infected with virus, but no new cases among students.

The state has started releasing coronavirus cases among Massachusetts college students, which it does on Wednesdays, along with data on town-by-town cases of the virus and high-risk communities.

The latest data comes as the number of Massachusetts communities that are now considered “high risk” for COVID spread increased to 40 on Wednesday.

Communities in the high risk category have had more than eight new daily cases per 100,000 residents over the last 14 days. As of Oct. 7, they include: Acushnet, Amherst, Attleboro, Avon, Boston, Brockton, Chelmsford, Chelsea, Dartmouth, Dracut, Dudley, Everett, Framingham, Haverhill, Holyoke, Hudson, Kingston, Lawrence, Leicester, Lowell, Lynn, Malden, Marlborough, Methuen, Middleton, Nantucket, New Bedford, North Andover, Plymouth, Randolph, Revere, Southborough, Southbridge, Springfield, Sunderland, Waltham, Webster, Winthrop, Woburn and Worcester.

DESE releases updated data on Thursdays.

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‘We don’t know why this is happening’: COVID-19 ‘long haulers’ show mysterious and sometimes debilitating symptoms months after being infected

40 Massachusetts communities now considered ‘high risk’ for COVID spread


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