The COVID-19 pandemic is adding an unanticipated concern for those affected by genetic diseases. Most of the new treatment achievements for these patients are made possible as a result of advances in viral-based products. Among them, adenoviruses (AdV) and especially adeno-associated viruses (AAV) are important players. The concerns and the conversation around this issue have increased as COVID-19 vaccines approach the market. What if the viral vectors become the mainstream strategy for vaccine development? Will the immune response elicited against the vector compromise the efficacy of future gene therapies? Patients with genetic diseases and patient advocacy groups are requesting information to the medical community about the potential impact of these vaccines in future gene therapy treatments, and physicians and scientists are not able to provide satisfactory answer yet. Importantly, the frequency of cross-reactivity among different AAV serotypes can be as high as 50%. This would have potential implications for patients with genetic disorders who could benefit from gene therapies, often coming in the form of AAV-based gene therapies. As in many other aspects, this pandemic is challenging our capacity to coordinate, plan ahead and align different medical objectives. In this case, having such conversation early on might allow us to make the right choices while we are still on time.
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http://dx.doi.org/10.1186/s13023-021-01958-3 | DOI Listing |
J Neurol
January 2025
Parkinson's Disease Research Clinic, Macquarie University, 75 Talavera Road, Sydney, NSW, 2109, Australia.
Impulse Control Disorders (ICDs) are increasingly recognized as a significant non-motor complication in Parkinson's disease (PD), impacting patients and their caregivers. ICDs in PD are primarily associated with dopaminergic treatments, particularly dopamine agonists, though not all patients develop these disorders, indicating a role for genetic and other clinical factors. Studies over the past few years suggest that the mesocorticolimbic reward system, a core neural substrate for impulsivity, is a key contributor to ICDs in PD.
View Article and Find Full Text PDFClin Transl Med
January 2025
Department of Dermatology and Allergy, University Hospital of Munich, Ludwig-Maximilian-University, Munich, Germany.
Background: Cancer immunotherapy has transformed metastatic cancer treatment, yet challenges persist regarding therapeutic efficacy. RECQL4, a RecQ-like helicase, plays a central role in DNA replication and repair as part of the DNA damage response, a pathway implicated in enhancing efficacy of immune checkpoint inhibitor (ICI) therapies. However, its role in patient response to ICI remains unclear.
View Article and Find Full Text PDFMed J Malaysia
January 2025
Klinik Kesihatan Seksyen 7, PKD Petaling, Ministry of Health Malaysia, Selangor, Malaysia.
Introduction: Cancer is one of the leading causes of morbidity and mortality worldwide. Breast cancer risk has risen due to lifestyle choices and genetic factors. Women with breast cancer symptoms experience lower quality of life (QoL), particularly in psychological and physical domains, compared to healthy women.
View Article and Find Full Text PDFSpine (Phila Pa 1976)
January 2025
Department of Research and Innovation, Division of Clinical Neuroscience, Oslo University Hospital, Oslo, Norway.
Study Design: Genome-wide association study (GWAS) meta-analysis with downstream analyses.
Objective: To explore the genetic architecture of chronic low back pain (cLBP) and identify underlying biological mechanisms that contribute to its development.
Summary Of Background Data: Chronic low back pain is prevalent and debilitating, with many cases having no identifiable biological cause.
Tunis Med
January 2025
University Hassan II of Casablanca, Faculty of medicine and pharmacy of Casablanca, Abderrahim HAROUCHI Mother-child hospital, Pediatric Anesthesiology and Intensive Care Unit, Laboratory of clinical immunology, inflammation and allergy (LICIA), Casablanca, Morocco.
Introduction: Pediatric sepsis remains a leading cause of morbidity and mortality in Africa. Nearly half of pediatric sepsis deaths occur in previously healthy children. The role of inborn errors of immunity (IEI) in susceptibility to sepsis is yet to be identified and their prevalence amongst previously healthy children admitted to the pediatric intensive care unit (PICU) is unclear.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!