CRISPR Gene Editing Leads to Improvements in Vision for People With Inherited Blindness, Clinical Trial Shows
Massachusetts Eye and Ear
The Mass Eye and Ear Eaton-Peabody Laboratory Associate Professor Zheng-Yi Chen speaks about his efforts to develop gene therapies for hearing loss and his recent success in rescuing hearing loss in aging mice
Glaucoma is a disease that damages the optic nerve, the cable that connects the eye to the brain. This can lead to blindness. There are currently no symptoms for early glaucoma, but later on patients may experience difficulty seeing in the dark or difficulty reading
Cultivated autologous limbal epithelial cells (CALEC) transplant, in which stem cells from the healthy eye and transplanted into the injured eye, for significant cornea injuries was found safe and led to gains in preliminary phase I clinical trial.
In research published last month in The Laryngoscope, Dr. Matthew Naunheim and the team at Mass Eye and Ear surveyed 1,522 people to explore three unanswered questions in laryngology.
Benjamin K. Mizell, MD, an anesthesiologist with a proven track record of leading major Mass Eye and Ear initiatives including its electronic medical record integration, has been named chief of Anesthesia at Mass Eye and Ear.
An international team of researchers led by Mass Eye and Ear discovered a new genetic mutation that leads to childhood glaucoma, and in the process uncovered a new mechanism for causing the disease.
Claes H. Dohlman, MD, Phd, considered a founder of modern cornea science, was awarded the 2022 António Champalimaud Vision Award for his vast contributions to vision research. The award comes with a €1,000,000 prize, one of the largest in scientific research.
An artificial-intelligence (AI) model built at Mass Eye and Ear was shown to be significantly more accurate than doctors at diagnosing pediatric ear infections in the first head-to-head evaluation of its kind. The model, called OtoDX, was more than 95 percent accurate in diagnosing an ear infection in a set of 22 test images compared to 65 percent accuracy among a group of clinicians who reviewed the same images.
Scientists have demonstrated that the APOE4 gene variant, which increases risk for Alzheimer’s but decreases risk of glaucoma in humans, blocks a disease cascade that leads to the destruction of retinal ganglion cells in glaucoma. Additionally, they showed in mouse models that the death of retinal ganglion cells – the cause of vision loss in glaucoma – can be prevented by using medications to inhibit a molecule called Galectin-3, which is regulated by the APOE gene. These findings taken together emphasize the critical role of APOE in glaucoma and suggest that Galectin-3 inhibitors hold promise as a glaucoma treatment, according to the authors.
Mass Eye and Ear researchers in the Eaton-Peabody Laboratories have been awarded a five-year, $12.5 million P50 Clinical Research Center Grant from the National Institute on Deafness and Other Communicable Disorders (NIDCD) of the National Institute of Health (NIH) to continue their research on cochlear synaptopathy, or hidden hearing loss, a type of hearing damage first discovered at Mass Eye and Ear in 2009. Funding from the grant extends support of four projects that aim to clarify the prevalence, nature and functional consequences of hidden hearing loss in humans.
Researchers from Mass Eye and Ear have developed a word-score model capable of estimating the amount of hidden hearing loss in human ears. This form of hearing loss is caused by cochlear nerve degeneration, and is not detected by standard audiogram tests.
Scientists from the Broad Institute of MIT and Harvard have developed a first-of-its-kind cross-tissue cell atlas, and in collaboration with researchers at Mass Eye and Ear, have uncovered new clues for specific cell types and genes involved in complex diseases. In a new study published May 12 in Science, researchers described for the first time how their novel cross-tissue cell atlas derived from an analysis of nuclei from 25 frozen samples from 8 tissue types may increase understanding of the cellular and genetic underpinnings of complex diseases, including heart disease and cancers.
A surgically implanted device that moves the tongue forward during sleep was found to safely and effectively reduce sleep apnea in adolescents with Down syndrome, according to a new study published April 21 in JAMA Otolaryngology – Head & Neck Surgery.
Mass Eye and Ear research shows the viral vector Anc80L65 efficiently transferred genetic cargo into the inner ear of nonhuman primates via a specialized surgical procedure, paving the way for a method that can be brought to clinical trials for hearing loss and vestibular disorder treatments.
New research shows liquid biopsy for HPV-associated head and neck cancer is more than 98% accurate and obtained a diagnosis 26 days quicker on average than conventional tissue biopsy, in addition to costing 38% less. With HPV-associated head and neck cancer rates on the rise, there is a great need for more accurate, less-invasive, faster and less expensive diagnostic tests.
Pernell T. Reid, MD, MHA, MS, an experienced health care administrator with nearly two decades of practice management and financial leadership experience in academic medicine, is joining Mass Eye and Ear (a member hospital of Mass General Brigham) as Senior Vice President of the Department of Otolaryngology–Head and Neck Surgery (OHNS).
Researchers have identified the mechanism that can lead to deafness in the rare syndrome, Norrie disease, which may lead to promising treatment targets for the incurable disease and other forms of profound hearing loss.
Laryngology researcher Kristina Simonyan, MD, PhD, Dr med, of Mass Eye and Ear, has been awarded an $11.9 million P50 Clinical Research Center Grant from the National Institute on Deafness and Other Communicable Disorders to form a new multidisciplinary center across four academic medical institutions that will be committed to conducting research on laryngeal dystonia and voice tremor, two debilitating neurological voice disorders.
Gene-based, single-dose AAVCOVID vaccine shown to offer disease protection in challenge study, and to elicit year-long immune response, according to new paper in Cell Host & Microbe.
A wearable computer vision device with vibrating wristbands was shown in a randomized trial to reduce collisions for both people who are blind or those who are visually impaired and using a long cane and/or guide dog by 37 percent, compared to using other mobility aids alone. Such technology may augment safety for independent travel for these individuals.
Mark Varvares, MD, FACS, has been appointed as the next Chief of the Departments of Otolaryngology-Head and Neck Surgery (OHNS) at Mass Eye and Ear and Massachusetts General Hospital (MGH) and as Chair of the Department of OHNS at Harvard Medical School (HMS), following an extensive year-long search process
Sleep disorders are associated with significantly higher rates of health care utilization, conservatively placing an additional $94.9 billion in costs each year to the U.S. health care system, according to a new study from researchers at Mass Eye and Ear, a member hospital of Mass General Brigham.
Michael S. Gilmore, PhD, a renowned microbiologist who has led infectious disease efforts at leading research institutions, has been appointed the first Chief Scientific Officer at Mass Eye and Ear, a member hospital of Mass General Brigham, following an extensive search.
In the largest genome-wide association study of glaucoma to date, an international team of researchers compared the genes of 34,179 people with the disease to 349,321 control subjects. They identified 127 genes linked to glaucoma, including 44 new gene loci and confirmed 83 previously reported loci.
The AAVCOVID vaccine program, a novel gene-based vaccine strategy that utilizes an adeno-associated virus (AAV) vector, was granted an award for up to $2.1 million from the Bill & Melinda Gates Foundation. In data released in preprint, two AAVCOVID vaccine candidates led to robust neutralizing antibody responses in mouse and nonhuman primate models from only a single dose. The candidates were also shown to be safe and well-tolerated in both models.
Dr. Mary Beth Cunnane, a radiologist who specializes in the imaging of patients with diseases of the eyes, ears, nose and head and neck, has been appointed Chief of Radiology at Mass Eye and Ear in Boston.
A new randomized controlled trial has found antibiotics are not necessary after routine sinus surgery for preventing infection and optimizing patient outcomes.
Proof-of-concept study represents first successful attempt to reverse the aging clock in animals through epigenetic reprogramming.
Hearing experts advocate in The New England Journal of Medicine for the FDA to implement the Over-the-Counter Hearing Aid Act of 2017. The FDA missed the deadline to release the much-anticipated regulations on August 18, 2020
Mass Eye and Ear is proud to announce the successful conclusion of its historic campaign, “Bold Science. Life-Changing Cures.” which raised $252M from philanthropy to advance research to treat and cure diseases of vision, hearing, and the head and neck. The campaign was led by co-chair Wyc Grousbeck, Boston Celtics CEO and Lead Owner and former Chairman of Mass Eye and Ear.
Researchers at Mass Eye and Ear have developed a unique diagnostic tool called DystoniaNet that uses artificial intelligence to detect dystonia from MRI scans in 0.36 seconds. DystoniaNet is the first technology of its kind to provide an objective diagnosis of the disorder. In a new study of 612 brain MRI scans, the platform diagnosed dystonia with 98.8 percent accuracy.
Mass Eye and Ear has received a five-year, $4 Million gift from Good Ventures to spur Dr. Joseph F. Arboleda-Velásquez's groundbreaking work into Alzheimer’s disease genetic resistance. This transformational gift will enable Dr. Arboleda-Velásquez to build upon the recent discovery of a genetic mutation that protected a woman who had a high familial risk for the condition and amyloid plaque deposits in her brain against early-onset Alzheimer’s.
Surgeons at Mass Eye and Ear have replaced the ocular surface of four patients who each experienced chemical burns to one eye by using their own stem cells taken from the other healthy eye, in a technique known as “cultivated autologous limbal epithelial cell transplantation” (CALEC). These four cases, all part of an ongoing clinical trial supported by the National Eye Institute of the NIH, represent the first procedures of their kind to occur in the United States.
In a new study of human ear tissues, hearing scientists have demonstrated that age-related hearing loss, also called presbycusis, is mainly caused by damage to hair cells, the sensory cells in the inner ear that transform sound-induced vibrations into the electrical signals that are relayed to the brain by the auditory nerve. Their research challenges the prevailing view of the last 60 years that age-related hearing loss is mainly driven by damage to the stria vascularis, the cellular “battery” that powers the hair cell’s mechanical-to-electrical signal conversion.
Massachusetts Eye and Ear, a member hospital of Mass General Brigham, is entering into an exclusive licensing agreement with Biogen to develop a potential treatment for inherited retinal degeneration due to mutations in the PRPF31 gene, which are among the most common causes for autosomal dominant retinitis pigmentosa.
Children who choke when they drink or eat may have what’s known as dysphagia, or a swallowing disorder -- one of the most common medical complaints seen in young children. A new study has found that by combining different medical disciplines in one center rather than a typical care journey making appointments one specialist at a time, children had better outcomes, reduced the number of procedures needed, and health care costs were reduced.
The AAVCOVID vaccine program at Massachusetts Eye and Ear and Massachusetts General Hospital has entered into manufacturing agreements with Viralgen, Aldevron and Catalent in order to support manufacturing of the experimental vaccine for clinical studies in the second half of 2020.
AveXis, will lead the manufacturing efforts of the new AAVCOVID vaccine, utilizing its cutting-edge AAV technology for treatment of rare and life-threatening neurological genetic diseases. AveXis has the option to manufacture the AAV vaccine for additional clinical development, registration and/or commercial activities.
The internationally-renowned Gene Therapy Program at the University of Pennsylvania is joining the AAVCOVID vaccine program led by Massachusetts Eye and Ear and Massachusetts General Hospital (MGH), members of Mass General Brigham. AAVCOVID is a unique gene-based vaccine candidate designed to protect against SARS-CoV-2, the virus that causes COVID-19.
The Emergency Medical Services (EMS) Committee of the Conference of Boston Teaching Hospitals is pleased to join our hospital Emergency Medicine colleagues, the American College of Emergency Physicians, the National Association of EMT’s, Governor Charles D. Baker and Lieutenant Governor Karyn Polito, and City of Boston Mayor Martin J. Walsh in celebrating National EMS Week 2020, commencing May 17th, 2020.
Massachusetts Eye and Ear and Massachusetts General Hospital (MGH), members of Mass General Brigham, today announced progress towards the testing and development of an experimental vaccine called AAVCOVID, a novel gene-based vaccine candidate against SARS-CoV2, the virus that causes COVID-19. The AAVCOVID Vaccine Program is a unique, gene-based vaccine strategy that uses adeno-associated viral (AAV) vector, a clinically established gene transfer technology leveraging the properties of a harmless viral carrier.
The Conference of Boston Teaching Hospitals' Executive Director, Patricia McMullin, released the following statement in response to President Trump’s Fiscal Year 2021 budget request, which proposes significant cuts to federal spending critical to teaching hospitals, including the National Institutes of Health, Medicaid, and graduate medical education.
A pair of biomarkers of brain function — one that represents “listening effort,” and another that measures ability to process rapid changes in frequencies — may help to explain why a person with normal hearing may struggle to follow conversations in noisy environments, according to a new study led by Massachusetts Eye and Ear researchers. The researchers hoped the study could inform the design of next-generation clinical testing for hidden hearing loss, a condition that cannot currently be measured using standard hearing exams.
Researchers have identified a novel cellular entry factor for adeno-associated virus vector (AAV) types—the most commonly used viral vectors for in vivo gene therapy.
Mass. Eye and Ear scientists report the identification of a new pathway linked to cell division in the ear. With this pathway, they were able to reprogram the inner ear’s cells to proliferate and regenerate hair cell-like cells in adult mouse models.
Researchers have identified a genetic mutation of the APOE Alzheimer’s disease gene that may provide protection against the neurological illness. The researchers hope this mutation may provide future prevention and treatment targets for Alzheimer’s and other neurodegenerative diseases.
Researchers from Massachusetts Eye and Ear, Harvard Medical School and a team of EPFL researchers have developed a conformable electrode implant that will allow people with a dysfunctional inner ear to hear again. This new technology would improve existing auditory brainstem implants, which have a number of shortcomings.
Massachusetts Eye and Ear raised more than 1.3 million at its 10th Annual Sense-ation! Gala on October 7, 2019, at the Westin Boston Waterfront. Guests were treated to a specially-arranged performance from cast members from Disney’s The Lion King, presented by Broadway In Boston.
Chronic conductive hearing loss, which can result from middle-ear infections, has been linked to speech recognition deficits, according to a new study led by scientists at Massachusetts Eye and Ear.