Therapeutics Commons^™

Safety And Feasibility Of Same-Admission Transcarotid Arterial Revascularization Prior To Heart Surgery For Patients Presenting With Concurrent, Severe Carotid Artery Stenosis, And Surgical Cardiac Disease, Brittany Landavazo, Matthew D. Kenny, Chandler Vernon, Nicolas Zea, David Nation, Bradley Boone, Jeff Apple, Kofi Quaye, Shir Yelovitch, Ryan S. Turley

HCA Healthcare Journal of Medicine

Introduction

Carotid artery stenosis and coronary artery disease are often co-morbid, with a prevalence of concurrent carotid and coronary artery stenosis approaching 50%. The optimal treatment for these patients has long been debated, with open carotid revascularization generally reserved for those with severe symptomatic carotid disease that precludes cardiac surgery. In this scenario, the role of less-invasive carotid artery stenting, particularly transcarotid arterial revascularization (TCAR), remains controversial and is not yet well studied. This study aims to present our outcomes and methodology for treating severe carotid stenosis with TCAR prior to cardiac surgery.

Methods

A retrospective chart review of the …

Epidemiology Of Healthcare-Associated Ventriculitis And Meningitis (Hcavm) And Community-Acquired Meningitis/Encephalitis And Evaluation Of An Off-Label Pcr Panel For Hcavm Diagnosis, Jaime Nassur, Katarina Schnell, Christine Dolon, Laura Walters, Devin Weber, Matthew Pettengill

Department of Medicine Faculty Papers

Healthcare-associated ventriculitis and meningitis (HCAVM) is associated with high morbidity and mortality, but rapid diagnostic tests intended to cover microorganisms that typically cause HCAVM are not commercially available. We sought to evaluate the etiology of HCAVM compared to community-acquired meningitis/encephalitis (CAME) at our healthcare system, and to study the utility of repurposing a multiplex molecular panel intended for positive blood cultures for HCAVM cases. Our epidemiologic review demonstrated that HCAVM cases were primarily caused by bacteria and fungi also associated with bloodstream infections, in contrast with CAME cases, which were primarily caused by herpesviruses and Cryptococcus. Eighty-two cerebrospinal fluid …

Controlled Antibiotic Release From Emulsion-Loaded Alginate And Fibrin Hydrogels Using Ultrasound, Ziba Ghareh Nazi Fam, Asia Winslow, Mario Fabiilli, Sam Varghese, Brian Oeffinger, Flemming Forsberg, Noreen Hickok, Lauren Delaney

Department of Radiology Faculty Papers

Postoperative infections remain a major complication after spinal fusion surgery, often caused by biofilm-forming bacteria that resist short-term antibiotic prophylaxis. Vancomycin (VAN) is sometimes delivered locally during surgery; however, levels diminish rapidly, leaving patients vulnerable to late-onset infections. We developed a composite hydrogel integrating perfluorohexane-based emulsions within alginate or fibrin matrices to enable both sustained and ultrasound-triggered VAN release. Water-in-oil-in-water emulsions were prepared using fluorosurfactant-stabilized perfluorohexane as the volatile oil phase, then embedded in hydrogels and exposed to ultrasound (2.5 MHz, 5.5 MPa peak negative pressure) to initiate acoustic droplet vaporization for triggered release. Drug release was quantified spectrophotometrically and …

When Unilateral Leg Swelling Is Not Deep Vein Thrombosis: A Case Of Disseminated Kaposi Sarcoma Revealing Advanced Human Immunodeficiency Virus, Ahmed Ismail, Zachary Wilson, Thomas Currow, Michael Dileonardo, Carsten Kirby

Department of Medicine Faculty Publications

Kaposi sarcoma (KS) is an acquired immunodeficiency syndrome (AIDS)-defining malignancy that can involve the skin, lymph nodes, and visceral organs in patients with advanced human immunodeficiency virus (HIV). We report the case of a 35-year-old man with no known prior medical history who presented to the charity care clinic complaining of recurrent thrush and two weeks of progressive right lower extremity swelling. His physical examination revealed painful, pruritic violaceous papules and plaques over the proximal right thigh that had been present for one week. He was referred to the emergency department (ED) for further workup. Venous duplex ultrasonography was negative …

P-15 Peptide Enhanced Bone Graft Improves Time To Fusion In Transforaminal Lumbar Interbody Fusion: A Randomized, Controlled, Investigational Device Exemption Study, James S. Harrop, Michael P. Steinmetz, John E. O'Toole, Christopher D Chaput, Rick C. Sasso, K. Brandon Strenge, Greg Maislin, Jeffrey P. Mullin, Thomas B. Freeman, Anthony Guanciale, Howard Lantner, Michael E. Janssen, David G. Schwartz, John M. Small, Wellington K. Hsu, Paul M. Arnold

Department of Neurosurgery Faculty Papers

STUDY DESIGN: Prospective, multicenter, single-blind, randomized, and controlled pivotal study.

OBJECTIVE: Compare time-to-fusion in patients treated with P-15L (PearlMatrix TM P-15 peptide enhanced bone graft) versus local autograft over 24 months and evaluate changes in pain and quality of life at 24 months relative to baseline.

SUMMARY OF BACKGROUND DATA: P-15L, an FDA-designated breakthrough device, is a composite bone graft with P-15, a 15-amino acid polypeptide that promotes cellular adhesion, proliferation, and differentiation to support bone formation.

METHODS: Patients (22-80 y) with degenerative disc disease were randomized to the investigational (P-15L) or control (local autograft) group during single-level transforaminal lumbar …

Pediatric Anesthesia And Virtual Reality: Managing Perioperative Pain And Anxiety– A Scoping Review, Moneer Sharafi, Mason Pham, Alec Wiscombe

Doctoral Projects

Pediatric surgery is common, but it often causes considerable anxiety and pain. A staggering percentage of children experience preoperative anxiety, which can heighten pain, delay recovery, and contribute to complications such as emergence delirium and longer-term behavioral issues (Fortier & Kain, 2015). Virtual reality (VR), which immerses users in computer-generated environments, has been proposed as a distraction technique to reduce these challenges. This Doctor of Nursing Practice (DNP) project conducted a scoping review to examine the effectiveness of VR in reducing perioperative pain and anxiety in pediatric patients. The review followed the Preferred Reporting Items for Systematic Reviews and Meta-Analyses …

Electrochemical Sensors For In Situ Monitoring Of Reactive Species During Cold Atmospheric Plasma-Based Therapies, Jonathan Thomas, Suneel Kumar, Gagana Karkada, Julia Sutter, Kristina Pattison, Jason Rainone, Dhruv Patel, Shashank Madhavan, Fred Krebs, Francois Berthiaume, Vandana Miller, Katharina Stapelmann

Kimmel Cancer Center Faculty Papers

Cold atmospheric pressure plasma (CAP) is emerging as a clinically relevant therapy for dermatological conditions such as actinic keratosis, warts, and chronic wounds. However, these therapies lack strategies to monitor CAP delivery in situ and to ensure delivery of an effective CAP dose without unwanted toxicity. CAP acts as a therapeutic agent in these biomedical applications primarily (but not solely) through reactive oxygen and nitrogen species (RONS) generated at transiently high local concentrations. Here we demonstrate the use of bio-electrochemical sensors capable of real-time measurements of key CAP RONS: hydrogen peroxide and oxidation-reduction-potential (ORP). In in vitro scratch assays and …

Editorial: Bridging Inflammation, Oxidative Stress, And Metabolic Dysfunction In Brain Disorders, Ansab Akhtar

School of Medicine Faculty Publications

We present this Special Issue of Brain Sciences, titled "Role of Inflammation, Oxidative Stress, and Metabolic Dysfunction in Neurodevelopmental and Neurodegenerative Diseases: Pharmacological Targets and Therapeutic Interventions", which brings together research and review articles that explore the molecular crosstalk between neuroinflammatory, oxidative stress, and metabolic pathways underlying both neurodegenerative and neurodevelopmental disorders [...].

Validation Of A Prognostic Nomogram For Locally Advanced Oropharyngeal Carcinoma Treated With Intensity-Modulated Radiotherapy With/Without Systemic Therapy, P. Bossi, R. Gili, S. Calza, L. Licitra, M. Maddalo, A. Bacigalupo, L. Belgioia, J. Cacicedo, N. Facchinetti, A. Garcia Castano, M. Oliva, P. Bonomo, G. Sanguineti, P. Franco, A. Argiris, A. Psyrri, E. Orlandi

Kimmel Cancer Center Faculty Papers

BACKGROUND: Head and neck squamous-cell carcinoma (HNSCC), particularly oropharyngeal cancer (OPC), exhibits diverse prognostic factors influenced by demographic and lifestyle variables. Prognostic nomograms, such as the one developed by Fakhry et al., incorporate clinical and biological markers to predict overall survival (OS) and progression-free survival (PFS) in OPC patients. However, their applicability to European populations remains unverified. This study aimed to externally validate Fakhry's prognostic nomogram in a large European cohort of locally advanced OPC patients treated with definitive intensity-modulated radiotherapy (IMRT) with or without systemic therapy.

METHODS: This is a retrospective external validation study conducted on 805 OPC patients …

The Science Of Sound: Studying The Cognitive, Emotional, And Physiological Effects Of Frequency, Genre, And Music, Jett Yarborough

Senior Honors Theses

Music influences emotion, physiology, and cognition, yet little is known about how the frequency it is tuned to affects these influences. Prior research has shown that music tuned to 432 Hz can reduce stress, lower blood pressure, and improve sleep. My colleagues and I conducted two studies to investigate this. Our research found that music tuned to 432 Hz promotes a significant increase in memory retention and induces a state of focus and relaxation. These results suggest that shifting from the standard tuning frequency of 440 Hz to 432 Hz could have a profoundly positive impact on our daily lives. …

Impact Of A Focused Best Practice Workshop On Athletic Trainer Confidence, Jaclyn B. Arduini, Jennifer L. Volberding, Zachary A. Murray

Clinical Practice in Athletic Training

Athletic trainers are required to be proficient in emergency management skills, without review these skills may decay creating a potentially dangerous knowledge gap. Confidence measures of emergency management skills were taken before and after a workshop to determine if they changed due to the review of material. All 11 confidence measures increased post workshop, seven significantly, including rectal temperature, hemostatic agents, external hemostatic modalities, in-line stabilization, measurement and application of a cervical collar, football and other equipment removal. ATs should aim to incorporate emergency management review into continuing education choices.

Incorporating Balance Training Into Ankle Sprain Injury Prevention Programs: An Evidence-To-Practice Review, David Tomchuk, Jacob Meyer, Kyle Schneider, Ben Fox

Clinical Practice in Athletic Training

Ankle sprains are among the most common injuries in sports and non-sporting activities. For patients, ankle sprains can result in multiple short and long-term hardships, such as financial burdens of evaluation and treatment, absenteeism from sport and physical activity, and diminished ankle and lower extremity function. However, athletic trainers and other stakeholders are also impacted by administrative and time burdens in caring for ankle sprains and how ankle sprains affect team dynamics and performance. The 2022 guiding systematic review evaluated the effectiveness of incorporating preventive balance training exercises on the incidence of ankle sprains in soccer athletes. The search performed …

Examining Burnout Among Athletic Trainers Employed In The College And Secondary School Settings, Stephanie M. Singe, Dayna Liu, Alexandrya Cairns

Clinical Practice in Athletic Training

Burnout continues to be an important concern for athletic trainers working traditional employment settings. The purpose of this study was to compare the secondary school and collegiate settings in relation to burnout. Both secondary school and collegiate athletic trainers participated in a self-reported, online cross-sectional design (Qualtrics) to collect data on their experiences with burnout. Data collection occurred during the second week of October 2023 and ceased during the early weeks of November. Collegiate athletic trainers reported moderate burnout overall (54.56 ±15.89) on the CBI whereas secondary school athletic trainers reported low burnout overall (46.67 ±16.57). On the personal-related burnout …

Use Of An Electronic Dynamometer Following Pitching To Identify Fatigue In Softball Pitchers, Bethany L. Hansberger, Peter Lisman

Clinical Practice in Athletic Training

This study used a case-series design to collect in-game fatigue measures for collegiate softball pitchers immediately after pitching during the competitive season. Five softball pitchers (age = 19.8 ± 1.5 years; height = 169.9±6.1 cm; body mass = 79.1±22.6 kg) from one Division I program were recruited. Isometric internal and external rotation, scaption, and grip strength measures were collected using an electronic dynamometer at the beginning and conclusion of the fall non-traditional season, and at the beginning of preseason in January. Strength measures were also obtained during the competitive season at the conclusion of pitching in a game and compared …

Concurrent Acl And Medial/Lateral Meniscal Repair In A Collegiate Soccer Player: Disablement Model Case Study, Rebecca L. Perry, Angelica Alicea, Jeanette Welch, Carl White, Emily E. Hildebrand, Peter Lisman

Clinical Practice in Athletic Training

This case examines a 19-year-old female Division I collegiate soccer player who underwent anterior cruciate ligament (ACL) reconstruction with medial and lateral meniscal repairs. Though meniscal tears often occur in conjunction with ACL injury, incidence of concurrent medial and lateral tears with presence of a displaced bucket-handle lesion is rare. This patient attempted to tackle an oncoming opponent in which contact caused forceful trunk rotation away from her planted right foot. Sideline evaluation revealed medial and posterior knee pain with active knee range of motion deficits. Further evaluation revealed apprehension and muscle guarding during Lachman’s, anterior drawer, and valgus stress …

Suturing Education For Athletic Trainers: An Evidence-To-Practice Review, Morgan K. Webb, Brett T. Heim, Allyson E. Ritchey, Kenya Moore, Zachary K. Winkelmann

Clinical Practice in Athletic Training

Teaching suturing skills to medical students is a critical component of their education. Implementing suturing into a comprehensive framework is developed from how professors teach the skill. Suturing is a required skill by the General Medical Council that all medical students must have, and how it is being introduced to new students is changing. This review aimed to summarize different educational interventions and determine the most efficient and cost-effective way to teach future suturing skills. The original systematic review included 25 articles in the final data synthesis. Fourteen studies were randomized control trials, six were quasi-experimental, and five were observational. …

Conservative Management Of Athletic Pubalgia In A Collegiate Beach Volleyball Athlete: A Case Study, Sydney F. Hendrick, Amy L. Fraley, Madison V. Kefferstan, Zachary K. Winkelmann

Clinical Practice in Athletic Training

A 24-year-old female collegiate beach volleyball athlete experienced sharp pain in the pubis symphysis region after turning and running to reach a ball hit over her right shoulder. The patient was unable to move her lower extremities without extreme pain and required help off the court. She was referred to the student health center, where no initial imaging was performed. Differential diagnoses included osteitis pubis, abdominal strain, and athletic pubalgia. Magnetic resonance imaging (MRI) showed a small partial sports hernia with the shearing of the aponeurotic plate. The team physician recommended conservative management. The patient requested a consultation with a …

Introducing Patient-Centered Outcomes To Preceptors In Caate-Approved Athletic Training Programs, K. Judge

Clinical Practice in Athletic Training

The 2020 standards set by the Commission on Accreditation of Athletic Training Education (CAATE) now require the incorporation of the International Classification of Function and Patient-Centered Outcomes into professional practice. These standards can be introduced initially through simulation, but to develop a competent and culturally aware professional, outcome measures should be completed during clinical experiences under the guidance of approved preceptors. As this is a relatively new standard, many preceptors did not receive this as part of their education and may be uncomfortable integrating outcomes into their practice. This lack of integration can negatively affect the learning opportunities of athletic …

Psychological And Psychosocial Effects Of Contemplative Practices In The Athletic Population: A Systematic Review, Victoria Baker, Emily Duckett, Alison Gardiner-Shires, Lindsey Keenan

Clinical Practice in Athletic Training

Mental health concerns in athletes are becoming a primary focus for institutions, companies, coaches, and more. An athlete carries an abundant load of stressors due to their lifestyle. The stressors can negatively impact an athlete by producing anxiety, depression, insomnia, and other mental health concerns. Previous studies have found a connection between the use of contemplative practices on psychological and psychosocial effects in athletes, creating an opportunity to evaluate the research of contemplative practices in the athletic population. 5, 7, 8, 13-16 A systematic review was conducted using nine electronic databases. Seven studies were included in this review, each assessing …

Neuromuscular Deficits Following An Isolated Pcl Tear In A Collegiate Lacrosse Player: A Case Study, Mckenna G. Witlin, Erin R. Mcmullan, Courtney M. Meyer, Amy L. Fraley

Clinical Practice in Athletic Training

This disablement model case study outlines a 21-year-old female collegiate lacrosse player attending a United States service academy who presented with a unique neuromuscular deficit following a conservative grade II PCL tear rehabilitation. Nearing the end of her rehabilitation protocol, she noticed difficulty with toe-off while running; however, she did not report to the athletic trainer due to the institution’s holiday break proximity. An unexpected transfer of care occurred when the patient returned after the holidays, and she was cleared for full participation upon return. Several days after the patient was cleared, she reported her ankle feeling “stuck.” Upon evaluation, …

Improving The Discussion Of Suicide Prevention: A Quality Improvement Project, Chelsea Reynolds, Alyssa Zeitouni, Kathryn Stephenson

Clinical Practice in Athletic Training

Suicide is currently the second leading cause of death among adolescents in the United States and the third leading cause of death in this age group worldwide.1,2 Suicide prevention is understandably a growing focus in pediatrics. Currently, at the Children’s Hospital Outpatient Clinic, that is staffed by pediatric resident physicians, suicidal ideation is screened for using a PHQ-9 screen. However, at the time this study was initiated and completed, the TEEN screen was utilized. After the screening is performed, regardless of type, it is up to the provider to address these findings in a safe and effective manner. The goal …

Oral Tranexamic Acid Use For Melasma Is Not Associated With Thromboembolism: Findings From A Multicenter Propensity Score–Matched Electronic Health Record Cohort, Tyler Hernandez, Kayla Penny, Nicholas Culotta

School of Medicine Faculty Publications

No abstract provided.

Enhancing Cataract Surgery Outcomes: Optimal Use Of Pre- And Post-Operative Eye Drops, Keith Skolnick M.D., Anu Valiaveedu

Mako: NSU Undergraduate Student Journal

Many preoperative and postoperative cataract patients struggle with comprehending the use of prescription medication as directed. Language barriers and low health literacy levels are major factors contributing to improper use of prescriptions. To increase patients comprehension, the Fort Lauderdale Eye Institute employed an educational intervention consisting of a live presentation and an instructional video. Results found that 44% of patients were hesitant to ask questions to clinical staff, 32% felt overwhelmed, and nearly 70% lacked confidence in using their prescribed eye drops. Following the intervention, 91% of patients reported increased confidence in their medications, and most indicated that the video …

The Effect Of Hcv On Methadone Dose During Pregnancy, Sarah Boudova, Neel S. Iyer, Danielle M. Tholey, Jonathan M. Fenkel, Rupsa C. Boelig

Department of Obstetrics and Gynecology Faculty Papers

Pregnancy is a time of high patient motivation to initiate treatment for opioid use disorder (OUD). Hepatic drug metabolism can be altered by pregnancy and hepatitis C virus (HCV) infection. We aimed to examine the impact of HCV during pregnancy on methadone dosing. Retrospective chart review of all pregnant patients with OUD admitted for initiation of methadone from 1/2020-6/2022. Associations were examined using Student's T-tests, chi-squared tests, Fisher's exact tests and univariate and multivariate linear regression. We identified 191 pregnancies initiated on methadone, of which 188 were screened for HCV. 111 (59.0%) were HCV Antibody (Ab)+, of whom 108 were …

Utility Of The Us Preventive Services Task Force For Preeclampsia Risk Assessment And Aspirin Prophylaxis, Thomas Mcelrath, Arun Jeyabalan, Arkady Khodursky, Alison Moe, Manfred Lee, Maneesh Jain, Laura Goetzl, Elizabeth Sutton, Pamela Simmons, George Saade, Antonio Saad, Luis Pacheco, Esther Park-Hwang, Antonina Frolova, Ebony Carter, Ai-Ris Collier, Daniel Kiefer, Vincenzo Berghella, Rupsa Boelig, Michal Elovitz, Cynthia Gyamfi-Bannerman, Joseph Biggio, Kara Rood, William Grobman, Carrie Haverty, Morten Rasmussen

Department of Obstetrics and Gynecology Faculty Papers

IMPORTANCE: The US Preventive Services Task Force (USPSTF) guidelines on preeclampsia risk assessment and aspirin prophylaxis (AP) have not been evaluated for clinical utility.

OBJECTIVE: To evaluate which characteristics in the USPSTF guidelines identify risk status and the association of preeclampsia risk with AP recommendations.

DESIGN, SETTING, AND PARTICIPANTS: This observational cohort study enrolled from July 2020 to March 2023 with data analysis performed from October to December 2024. Enrollment occurred at 11 centers throughout the US or via direct-to-participant recruitment. Pregnant participants aged 18 years or older with a singleton pregnancy less than 22 weeks' gestation were selected via …

Current Trends In Clinical Trials For Merkel Cell Carcinoma (Mcc), Tilak Patel, Rachel Butz, Brian Boulmay, Vilija Vaitaitis

School of Medicine Faculty Publications

Merkel cell carcinoma (MCC) is a rare, aggressive neuroendocrine cutaneous malignancy with high rates of recurrence and metastasis. The treatment of MCC has historically involved surgery, radiation, and chemotherapy; however, current clinical trials are investigating immunotherapies, intratumoral injections, and combination therapies to improve outcomes. This review explores the current trends in clinical trials for MCC, highlighting recent advances in management.

Optimizing Stereotactic Intracranial Neoplasm Treatment: A Systematic Review Of Pet Integration With Gamma Knife Radiosurgery, Robert C. Subtirelu, Eric M. Teichner, Milo Writer, Kevin Bryan, Shiv Patil, Talha Khan, Lancelot Herpin, Raj N. Patel, Emily Christner, Chitra Parikh, Thomas Werner, Abass Alavi, Mona-Elisabeth Revheim

SKMC Student Presentations and Publications

OBJECTIVE: Traditional imaging modalities for the planning of Gamma Knife radiosurgery (GKRS) are non-specific and do not accurately delineate intracranial neoplasms. This study aimed to evaluate the utility of positron emission tomography (PET) for the planning of GKRS for intracranial neoplasms (ICNs) and the post-GKRS applications of PET for patient care.

METHODS: PubMed, Scopus, and ScienceDirect were searched in order to assemble relevant studies regarding the uses of PET in conjunction with GKRS for ICN treatment. PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines were followed to identify relevant studies on the use of PET in conjunction with …

Mesenchymal Stem Cell-Derived Extracellular Vesicles: Seeking Into Cell-Free Therapies For Bone-Affected Lysosomal Storage Disorders, Andrés Felipe Leal, Harry Pachajoa, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Lysosomal storage disorders (LSDs) constitute a group of monogenic systemic diseases resulting from deficiencies in specific lysosomal enzymes that cause the intralysosomal accumulation of non- or partially degraded substrates, leading to lysosomal dysfunction. In some cases of LSDs, the bone is more severely affected, thus producing skeletal manifestations in patients. Current therapies, such as enzyme replacement therapy (ERT) and gene therapy (GT), show limited efficacy in correcting skeletal abnormalities. Increasing evidence suggests that microenvironmental disturbances also contribute significantly to disease pathogenesis. Therefore, therapeutic strategies targeting lysosomal dysfunction and microenvironmental dysregulation are needed. Mesenchymal stem-cell-derived extracellular vesicles (MSC-EVs) are emerging as …

Brivaracetam For Spinal Cord Injury-Related Neuropathic Pain: Results Of A Pilot Double-Blinded, Randomized, Placebo-Controlled Clinical Trial, Leslie R. Morse, Ricardo A. Battaglino, Nguyen Nguyen, Brian Devries, Abigail Welch, Ana Lucia Battaglino, Clas Linnman, Michael Stillman, Robert Wudlick, Joda Glossner, Grant Anderton, Richard Goldstein, Scott P. Falci

Department of Medicine Faculty Papers

OBJECTIVE: Assess feasibility, safety, and tolerability of 3 months of oral brivaracetam for severe (9-10/10) spinal cord injury-related neuropathic pain.

METHODS: We conducted a multisite pilot study where adults were randomized to 3 months of brivaracetam (100 mg BID) or placebo. Pain was assessed via the Brief Pain Inventory, the International Spinal Cord Injury Pain Basic Dataset (version 2.0), and a daily pain diary.

RESULTS: We enrolled 24 participants across 2 sites and 14 participants were included in the final analysis (n = 8 placebo, n = 6 brivaracetam). The drug was tolerated with expected, nonserious side effects (nausea if …

Evaluation Of Aav Vectors With Tissue-Specific Or Ubiquitous Promoters In A Mouse Model Of Mucopolysaccharidosis Type Iva, Shaukat A. Khan, Jose Victor Álvarez, F. N. U. Nidhi, Eliana Benincore-Florez, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis type IVA (MPS IVA) is caused by a deficiency of N-acetyl-galactosamine-6-sulfate sulfatase (GALNS), leading to the accumulation of keratan sulfate and chondroitin-6-sulfate and development of severe skeletal dysplasia. Enzyme replacement therapy and hematopoietic stem cell transplantation are current treatment options but have limited impact on bone lesions. In this study, we investigated adeno-associated virus (AAV)8 or AAV9 vectors with liver-specific thyroxine-binding globulin or liver-specific promoter-a modification of hAAT (LSPX), liver-muscle tandem (LMTP), liver-bone tandem (LBTP), and ubiquitous cytomegalovirus early enhancer/chicken β-actin (CAG) promoters in MPS IVA mice to compare therapeutic efficacy on biochemical markers and bone pathology. All vectors …