Mass General’s Commitment to Expert Care
Massachusetts General Hospital (MGH) is a premier teaching hospital of Harvard Medical School. One of the cornerstones of MGH’s commitment to improving healthcare is by pushing the boundaries of medical research. The Mass General Research Institute is the largest hospital-based research program in the US, with over $1 billion in annual research operations and over 9,500 researchers working across more than 30 departments. MGH is dedicated to offering expert, safe, and effective patient care to the diverse communities it serves.
Gene Therapy for Mucolipidosis IV: A New Study
Mucolipidosis IV (MLIV) is an extremely rare neurologic disease of childhood with high unmet needs. Recently, co-corresponding authors Yulia Grishchuk, PhD, and Fengfeng Bei, PhD, published a new study in Molecular Therapy – Methods and Clinical Development. In the study, a novel AAV-vector with an enhanced ability to cross the blood-brain barrier was used as a gene supplementation approach. The authors found significant and dose-dependent improvement of neurologic function in the knockout animals treated with the AAV-CPP16 vector expressing the human MCOLN1 gene. Moreover, these treatment benefits were observed in the animals treated after the onset of neurologic dysfunction, suggesting successful recovery of neurologic function. AAV-mediated gene therapy shows promise as a therapeutic strategy for MLIV and warrants speedy translation into clinic.
The Importance of Collaboration
At Mass General, the brightest minds in medicine collaborate on behalf of patients to bridge innovative science with state-of-the-art clinical medicine. This culture of collaboration has helped MGH remain at the forefront of medicine.
Expert Care, In-Person or Virtually
MGH is dedicated to providing expert care to patients both in-person and virtually. Patients can schedule a visit to MGH that meets their needs, whether in-person or through telehealth.
A Commitment to Diverse Communities
MGH is committed to serving diverse communities and providing accessible healthcare. By partnering with community organizations and offering translation services and financial assistance, MGH aims to remove barriers to healthcare for all patients.
Alternative Medicine: An Overview
Alternative medicines refer to practices outside of conventional Western medicine. Alternative medicine can be broadly categorized into natural products, mind-body practices, and other complementary health approaches. Below, we will provide a brief overview of these categories.
Natural Products
Natural products refer to substances found in nature that are used for medicinal purposes. Examples include herbal medicines, vitamins, minerals, and probiotics. While these products are often marketed as safe and effective, it is important to note that many of them have not undergone rigorous testing and may have harmful side effects or interact with conventional medications.
Mind-Body Practices
Mind-body practices focus on the connection between the mind, body, and spirit. Examples include meditation, yoga, acupuncture, and chiropractic care. These practices are often used to manage stress, pain, and other conditions. While there is some evidence to suggest that mind-body practices can be beneficial, more research is needed to fully understand their effectiveness.
Complementary Health Approaches
Complementary health approaches are therapies that are used in conjunction with conventional Western medicine. Examples include massage therapy, hypnosis, and aromatherapy. While these therapies are often used to manage pain and other conditions, there is limited evidence to suggest their effectiveness.
In conclusion, alternative medicines are a diverse group of therapies outside of conventional Western medicine. While some alternative medicines may be effective in managing certain conditions, more research is needed to fully understand their benefits and potential risks.
Originally Post From https://www.massgeneral.org/news/research-spotlight/gene-therapy-improves-brain-functions-mucolipidosis-type-iv
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