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Researchers at Children’s Hospital of Philadelphia Show Gene Therapy Can Improve Quality of Life for Companion Dogs with Hemophilia
In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.
Children’s Hospital of Philadelphia Preclinical Study Demonstrates Improved Gene Therapy Approach to Treating Metachromatic Leukodystrophy
In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment for metachromatic leukodystrophy (MLD), a rare disease in young children characterized by the deficient activity of a critical enzyme. Without effective treatment, MLD leads to rapidly declining neurological skills in young children and is potentially fatal. The promising findings were published in the journal Molecular Therapy Nucleic Acids.
Children’s Hospital of Philadelphia Preclinical Study Unveils Promising New Treatment Approach for Fanconi Anemia Patients
In a preclinical study, researchers at Children’s Hospital of Philadelphia (CHOP) demonstrated a novel treatment strategy for patients with Fanconi anemia (FA), a rare genetic disease resulting in loss of blood-forming stem cells in the bone marrow. The researchers successfully used in situ mRNA delivered directly into the body by lipid nanoparticles (LNPs) to target bone marrow cells and transiently restore stem cell function in patients with FA.
First of Its Kind Gene Therapy Model Offers Hope for X-Linked Sideroblastic Anemia Treatment
Researchers at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine pioneered a first of its kind gene therapy model that offers a potential breakthrough in treating X-linked sideroblastic anemia (XLSA), a rare congenital anemia caused by mutations in the ALAS2 gene crucial for the synthesis of heme, a key compound in hemoglobin. This study marks the first time researchers studied gene therapy to treat this disease, which the authors underscore could have an impact on a broad spectrum of diseases. The research was published today and featured on the cover of the journal Blood.
Beyond Blood Fall 2024 Newsletter
Family Newsletter from the Comprehensive Hemostasis and Thrombosis Center at Children’s Hospital of Philadelphia.
Children’s Hospital of Philadelphia Researchers Find Hemophilia A Drug May Be Effective for Select People with Hemophilia B
Researchers at Children’s Hospital of Philadelphia (CHOP) found that emicizumab, a widely used drug for hemophilia A, could also improve blood clotting for select people with hemophilia B depending on their genes. The preclinical study, recently published in Blood, offers proof that this class of hemophilia A drug could potentially be used to treat hemophilia B.
Children’s Hospital of Philadelphia Researchers Find Cytokine Panels Informed Clinical Management in Nearly 30% of Pediatric Cases
The findings show how more rapid results can improve practice and lead to more accurate and timely diagnoses
Children’s Hospital of Philadelphia Leader in Sickle Cell Disease Elected to National Academy of Medicine
Alexis A. Thompson, MD, MPH, chief, Division of Hematology, and the Elias Schwartz MD Endowed Chair in Hematology at CHOP, has been recognized for her leadership in sickle cell disease (SCD) treatment and research.
First Patients at CHOP Begin Newly Approved Sickle Cell Gene Therapies
CHOP announced that its first patients outside of clinical trials have begun the treatment process for the newly FDA approved sickle cell gene therapies.
CHOP Researchers Develop Innovative Model Offering New Insights to Improve and Overcome Current Obstacles in Hemophilia A Gene Therapy
Researchers at CHOP announced the results of a new study offering insights into the development of next-generation gene therapies to treat hemophilia A.