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2021

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Articles 3091 - 3120 of 4713

Full-Text Articles in Medical Specialties

C33: Covid Encephalitis Resulting In Focal Seizures With Subsequent Changes On Mri Imaging, Adena Shahinian Apr 2021

C33: Covid Encephalitis Resulting In Focal Seizures With Subsequent Changes On Mri Imaging, Adena Shahinian

Annual Research Symposium

No abstract provided.


C30: A Comparison Of Cbct Soft Tissue With Bellus3d Facial Scan Superimposition, Gerald Sherrick Apr 2021

C30: A Comparison Of Cbct Soft Tissue With Bellus3d Facial Scan Superimposition, Gerald Sherrick

Annual Research Symposium

Research Symposium


C29: Epilepsy Partialis Continua With Transient Post-Ictal Changes On Mri, Trusha Mehta Do Apr 2021

C29: Epilepsy Partialis Continua With Transient Post-Ictal Changes On Mri, Trusha Mehta Do

Annual Research Symposium

No abstract provided.


C28: Acute Encephalopathy As A Presenting Sign Of Suspected Lupus Cerebritis, Jasmine Chopra Apr 2021

C28: Acute Encephalopathy As A Presenting Sign Of Suspected Lupus Cerebritis, Jasmine Chopra

Annual Research Symposium

No abstract provided.


C27: A Case Of Epstein-Barr Virus As Rare Suspected Etiology Of Rhombencephalitis, Devin Bradshaw, Adena Shahinian Do, Trusha Mehta Do, Jason Smuckler Do, Mckenzie Merrit Do, Paul Janda Do, Jd, Jay Mahajan Do, Mark Mckenzie Do Apr 2021

C27: A Case Of Epstein-Barr Virus As Rare Suspected Etiology Of Rhombencephalitis, Devin Bradshaw, Adena Shahinian Do, Trusha Mehta Do, Jason Smuckler Do, Mckenzie Merrit Do, Paul Janda Do, Jd, Jay Mahajan Do, Mark Mckenzie Do

Annual Research Symposium

No abstract provided.


C25: The “Spot Sign”: A Predictor Of Hematoma Expansion, Mckenzie Merritt Apr 2021

C25: The “Spot Sign”: A Predictor Of Hematoma Expansion, Mckenzie Merritt

Annual Research Symposium

No abstract provided.


C22: Eravacycline Versus Best Previously Available Therapy In Adults With Pneumonia Due To Drug-Resistant Acinetobacter Baumannii, Courtney Jane Scott, Rebecca Jayakumar, Elizabeth Zhu, Velliyur Viswesh Apr 2021

C22: Eravacycline Versus Best Previously Available Therapy In Adults With Pneumonia Due To Drug-Resistant Acinetobacter Baumannii, Courtney Jane Scott, Rebecca Jayakumar, Elizabeth Zhu, Velliyur Viswesh

Annual Research Symposium

There are no published studies documenting outcomes with the use of eravacycline against drug-resistant Acinetobacter baumannii. The purpose of this study is to assess the efficacy of eravacycline use, in comparison to best previously available therapy, in patients with drug-resistant Acinetobacter baumannii pneumonia.


C21: Diagnostic Accuracy Of Rapid Antigen Tests For Covid-19 Compared To The Viral Genetic Test In Adults: A Systematic Review Protocol, Ellyn Hirabayashi, Shelby Feliciano Apr 2021

C21: Diagnostic Accuracy Of Rapid Antigen Tests For Covid-19 Compared To The Viral Genetic Test In Adults: A Systematic Review Protocol, Ellyn Hirabayashi, Shelby Feliciano

Annual Research Symposium

No abstract provided.


C17: Programmed Cell Death Ligand- 1 Expression As A Prognostic Indicator In Oral Squamous Cell Carcinoma, Dr. Samar Saeed Khan Apr 2021

C17: Programmed Cell Death Ligand- 1 Expression As A Prognostic Indicator In Oral Squamous Cell Carcinoma, Dr. Samar Saeed Khan

Annual Research Symposium

No abstract provided.


C16: On-The-Spot Transport Media For The Freshly Biopsied Soft Tissue Specimen: A Comparative Study, Snehashish Ghosh Apr 2021

C16: On-The-Spot Transport Media For The Freshly Biopsied Soft Tissue Specimen: A Comparative Study, Snehashish Ghosh

Annual Research Symposium

No abstract provided.


C8: Intravenous (Iv) Bolus Versus Iv Infusion As Initial Approach For Blood Pressure (Bp) In Acute Ischemic Stroke (Ais) Patients Receiving Tissue-Plasminogen Activator (Tpa), Alexander D. Bonca, Alana Whittaker Apr 2021

C8: Intravenous (Iv) Bolus Versus Iv Infusion As Initial Approach For Blood Pressure (Bp) In Acute Ischemic Stroke (Ais) Patients Receiving Tissue-Plasminogen Activator (Tpa), Alexander D. Bonca, Alana Whittaker

Annual Research Symposium

No abstract provided.


C4: Comparing The Sub-Gingival Levels Of Cytomegalovirus, Epstein-Barr Virus, Porphyromonas Gingivalis In Human Immunodeficiency Virus-1 Seropositive Patients With And Without Antiretroviral Therapy, Supriya Kheur Apr 2021

C4: Comparing The Sub-Gingival Levels Of Cytomegalovirus, Epstein-Barr Virus, Porphyromonas Gingivalis In Human Immunodeficiency Virus-1 Seropositive Patients With And Without Antiretroviral Therapy, Supriya Kheur

Annual Research Symposium

No abstract provided.


S14: Factors Affecting Dental Students’ Comfort With Online Synchronous Learning, Roberto Lara Ms, Olivia R. Kalloo Ms, Mariana Pavlova Dmd, Donna Kritz-Silverstein Phd, David G. Mcmillan Dds Apr 2021

S14: Factors Affecting Dental Students’ Comfort With Online Synchronous Learning, Roberto Lara Ms, Olivia R. Kalloo Ms, Mariana Pavlova Dmd, Donna Kritz-Silverstein Phd, David G. Mcmillan Dds

Annual Research Symposium

Background: The COVID-19 pandemic has caused many universities to expand the use of videoconferencing technology to continue academic coursework. This study examines dental students’ experience with videoconferencing technology, comfort levels, and perceptions of using videoconferencing technology.

Methods: 54 out of 100 second-year dental students enrolled in a course on local anesthesia, 54 completed a survey following an online synchronous lecture given in September 2020. Survey questions asked about prior experience with videoconferencing, comfort levels, and reasons for not turning on their video (showing their face).

Results: Overall, 48.2% had little or no prior experience with videoconferencing prior to March 2020. …


S12: Assessing Ambulatory Care Pharmacist Interest In Scholarly Activities, Christopher O. Hernandez Apr 2021

S12: Assessing Ambulatory Care Pharmacist Interest In Scholarly Activities, Christopher O. Hernandez

Annual Research Symposium

No abstract provided.


S8: The Implementation Of Telehealth Scenarios And Covid-19 Training To Prepare Nurses For The Future Of Healthcare, Shamar Lejardi, Kiley Anders, Georgene Kreger, Aja James, Bethany Haward, Natalie Maughan, Courtney Gormus, Jessica Powell, Jene Hurlbut, Imelda Revuelto Apr 2021

S8: The Implementation Of Telehealth Scenarios And Covid-19 Training To Prepare Nurses For The Future Of Healthcare, Shamar Lejardi, Kiley Anders, Georgene Kreger, Aja James, Bethany Haward, Natalie Maughan, Courtney Gormus, Jessica Powell, Jene Hurlbut, Imelda Revuelto

Annual Research Symposium

No abstract provided.


S3: Post-Covid-19 Recovery Care Plan For Older Adults With Continuing Symptoms Of Breathlessness And Fatigue, Nancy M. Bryan, Marie Wright-Rolf Edd, Msn, Rn, Siboney Zelaya Ph.D., Mph, Msn Ed, Rn, Justin Carbonel Apr 2021

S3: Post-Covid-19 Recovery Care Plan For Older Adults With Continuing Symptoms Of Breathlessness And Fatigue, Nancy M. Bryan, Marie Wright-Rolf Edd, Msn, Rn, Siboney Zelaya Ph.D., Mph, Msn Ed, Rn, Justin Carbonel

Annual Research Symposium

Background:

The CDC research actively continues gathering information related to short and long-term symptoms that remain following the acute phase of Covid-19 diagnosis (CDC, 2020).

Fatigue and shortness of breath are listed among the most commonly reported long-term symptoms of Covid-19:

  1. Fatigue
  2. Shortness of Breath
  3. Cough
  4. Joint Pain
  5. Chest Pain (CDC, 2020).

Evidence suggests that older adults over the age of 60 who have multiple comorbidities are more susceptible and are at a higher risk of contracting the Covid-19 virus thus potentially prolonging recovery time post diagnosis (Greenhalgh, Knight, A’Court, Buxton, & Husain, 2020).

The Agency for Healthcare Research and …


P14: Emerging Role Of Pharmacists In Treatment And Vaccination For Covid-19, Dwaynie Bacon Jr, Man Ha, Dr. Mandal Apr 2021

P14: Emerging Role Of Pharmacists In Treatment And Vaccination For Covid-19, Dwaynie Bacon Jr, Man Ha, Dr. Mandal

Annual Research Symposium

No abstract provided.


P13: Demographic Characteristics Of Hpv Infections And Reporting Among Women In The United States, Jake Tuft Apr 2021

P13: Demographic Characteristics Of Hpv Infections And Reporting Among Women In The United States, Jake Tuft

Annual Research Symposium

No abstract provided.


P5: Covid-19 Vaccine: Technology Platform, Efficacy And Immune Protection, Manas Mandal Apr 2021

P5: Covid-19 Vaccine: Technology Platform, Efficacy And Immune Protection, Manas Mandal

Annual Research Symposium

Roseman Research Day Symposium.


P2: Characteristics Of Individuals With Oral Hpv Positive Results, Val Cheever, Eric Hon, Andrew J. Gross, Man Hung Apr 2021

P2: Characteristics Of Individuals With Oral Hpv Positive Results, Val Cheever, Eric Hon, Andrew J. Gross, Man Hung

Annual Research Symposium

No abstract provided.


O2: In-Silico Approach To Analyze Anticancer Activity Of Withania Somnifera In Oral Cancer, Gauri Kumbhar Apr 2021

O2: In-Silico Approach To Analyze Anticancer Activity Of Withania Somnifera In Oral Cancer, Gauri Kumbhar

Annual Research Symposium

No abstract provided.


Hypertension Management: Improving Medication Adherence Through The Use Of Daily Text Messages, Gaelle Bulabula Apr 2021

Hypertension Management: Improving Medication Adherence Through The Use Of Daily Text Messages, Gaelle Bulabula

Evidence-Based Practice Project Reports

Hypertension (HTN) is the leading cause of hospitalization and is responsible for approximately 50% of cardiovascular and cerebrovascular events (Al-Noumani, et al., 2018). Uncontrolled HTN can lead to comorbidities including ischemic heart disease, renal disease, or life-threatening arrythmias (World Health Organization [WHO], 2019). The leading cause of HTN is medication non-compliance (Varleta, et al., 2017). The purpose of this evidence-based practice project was to determine the effectiveness of daily text messages on antihypertensive medication adherence. A comprehensive review of literature generated ten high-level evidence studies demonstrating that daily text messages near each medication dosing time is best practice for improving …


Efficacy And Safety Of Dotinurad In Hyperuricemic Patients With Or Without Gout: A Systematic Review And Meta-Analysis Of Randomized Controlled Trials, Ayman Iqbal, Kinza Iqbal, Eisha Farid, Ali Ishaque, Muhammad Hasanain, Taha Bin Arif, Shajeea Arshad Ali, Sawai Singh Rathore, Mehreen Malik Apr 2021

Efficacy And Safety Of Dotinurad In Hyperuricemic Patients With Or Without Gout: A Systematic Review And Meta-Analysis Of Randomized Controlled Trials, Ayman Iqbal, Kinza Iqbal, Eisha Farid, Ali Ishaque, Muhammad Hasanain, Taha Bin Arif, Shajeea Arshad Ali, Sawai Singh Rathore, Mehreen Malik

Department of Anaesthesia

Introduction: A systematic review and meta-analysis of the available randomized controlled trials (RCTs) were conducted to investigate the efficacy and safety of dotinurad in hyperuricemic patients with or without gout. Dotinurad is a novel selective urate reabsorption inhibitor (SURI) that increases uric acid excretion by selectively inhibiting urate transporter 1 (URAT1). To the best of our knowledge, this is the first meta-analysis conducted to gauge the efficacy and safety of dotinurad.
Methods: Electronic databases (PubMed, the Cochrane Library, and ClinicalTrials.gov) were searched from inception till March 2, 2021, according to the Preferred Reporting Items for Systematic Review and Meta-Analysis …


Nbtxr3 Radiotherapy-Activated Functionalized Hafnium Oxide Nanoparticles Show Efficient Antitumor Effects Across A Large Panel Of Human Cancer Models., Ping Zhang, Julie Marill, Audrey Darmon, Naeemunnisa Mohamed Anesary, Bo Lu, Md, Sébastien Paris Apr 2021

Nbtxr3 Radiotherapy-Activated Functionalized Hafnium Oxide Nanoparticles Show Efficient Antitumor Effects Across A Large Panel Of Human Cancer Models., Ping Zhang, Julie Marill, Audrey Darmon, Naeemunnisa Mohamed Anesary, Bo Lu, Md, Sébastien Paris

Department of Radiation Oncology Faculty Papers

Purpose: The side effects of radiotherapy induced on healthy tissue limit its use. To overcome this issue and fully exploit the potential of radiotherapy to treat cancers, the first-in-class radioenhancer NBTXR3 (functionalized hafnium oxide nanoparticles) has been designed to amplify the effects of radiotherapy.

Patients and Methods: Thanks to its physical mode of action, NBTXR3 has the potential to be used to treat any type of solid tumor. Here we demonstrate that NBTXR3 can be used to treat a wide variety of solid cancers. For this, we evaluated different parameters on a large panel of human cancer models, such as …


Hydronephrosis Classifications: Has Utd Overtaken Apd And Sfu? A Worldwide Survey., Santiago Vallasciani, Anna Bujons Tur, John Gatti, Marcos Machado, Christopher S. Cooper, Marie Klaire Farrugia, Huixia Zhou, Mohammed El Anbari, Pedro-José Lopez Apr 2021

Hydronephrosis Classifications: Has Utd Overtaken Apd And Sfu? A Worldwide Survey., Santiago Vallasciani, Anna Bujons Tur, John Gatti, Marcos Machado, Christopher S. Cooper, Marie Klaire Farrugia, Huixia Zhou, Mohammed El Anbari, Pedro-José Lopez

Manuscripts, Articles, Book Chapters and Other Papers

Objective: To collect baseline information on the ultrasonographic reporting preferences.

Method: A 13-multiple choice questionnaire was designed and distributed worldwide among pediatric urologists, pediatric surgeons, and urologists. The statistical analysis of the survey data consisted of 3 steps: a univariate analysis, a bivariate and a multivariate analysis.

Results: Three hundred eighty participants responded from all the continents. The bivariate analysis showed the significant differences in the geographical area, the years of experience and the volume of cases. Most of the physicians prefer the SFU and APD systems because of familiarity and simplicity (37 and 34%, respectively). Respondents noted that their …


Functional Interpretation Of Atad3a Variants In Neuro-Mitochondrial Phenotypes, Zheng Yie Yap, Yo Han Park, Saskia B Wortmann, Adam C Gunning, Shlomit Ezer, Sukyeong Lee, Lita Duraine, Ekkehard Wilichowski, Kate Wilson, Johannes A Mayr, Matias Wagner, Hong Li, Usha Kini, Emily Davis Black, Kristin G Monaghan, James R Lupski, Sian Ellard, Dominik S Westphal, Tamar Harel, Wan Hee Yoon Apr 2021

Functional Interpretation Of Atad3a Variants In Neuro-Mitochondrial Phenotypes, Zheng Yie Yap, Yo Han Park, Saskia B Wortmann, Adam C Gunning, Shlomit Ezer, Sukyeong Lee, Lita Duraine, Ekkehard Wilichowski, Kate Wilson, Johannes A Mayr, Matias Wagner, Hong Li, Usha Kini, Emily Davis Black, Kristin G Monaghan, James R Lupski, Sian Ellard, Dominik S Westphal, Tamar Harel, Wan Hee Yoon

Faculty, Staff and Students Publications

BACKGROUND: ATPase family AAA-domain containing protein 3A (ATAD3A) is a nuclear-encoded mitochondrial membrane-anchored protein involved in diverse processes including mitochondrial dynamics, mitochondrial DNA organization, and cholesterol metabolism. Biallelic deletions (null), recessive missense variants (hypomorph), and heterozygous missense variants or duplications (antimorph) in ATAD3A lead to neurological syndromes in humans.

METHODS: To expand the mutational spectrum of ATAD3A variants and to provide functional interpretation of missense alleles in trans to deletion alleles, we performed exome sequencing for identification of single nucleotide variants (SNVs) and copy number variants (CNVs) in ATAD3A in individuals with neurological and mitochondrial phenotypes. A Drosophila Atad3a Gal4 …


Proteogenomic And Metabolomic Characterization Of Human Glioblastoma, Liang-Bo Wang, Alla Karpova, Marina A Gritsenko, Jennifer E Kyle, Song Cao, Yize Li, Dmitry Rykunov, Antonio Colaprico, Joseph H Rothstein, Runyu Hong, Vasileios Stathias, Macintosh Cornwell, Francesca Petralia, Yige Wu, Boris Reva, Karsten Krug, Pietro Pugliese, Emily Kawaler, Lindsey K Olsen, Wen-Wei Liang, Xiaoyu Song, Yongchao Dou, Michael C Wendl, Wagma Caravan, Wenke Liu, Daniel Cui Zhou, Jiayi Ji, Chia-Feng Tsai, Vladislav A Petyuk, Jamie Moon, Weiping Ma, Rosalie K Chu, Karl K Weitz, Ronald J Moore, Matthew E Monroe, Rui Zhao, Xiaolu Yang, Seungyeul Yoo, Azra Krek, Alexis Demopoulos, Houxiang Zhu, Matthew A Wyczalkowski, Joshua F Mcmichael, Brittany L Henderson, Caleb M Lindgren, Hannah Boekweg, Shuangjia Lu, Jessika Baral, Lijun Yao, Kelly G Stratton, Lisa M Bramer, Erika Zink, Sneha P Couvillion, Kent J Bloodsworth, Shankha Satpathy, Weiva Sieh, Simina M Boca, Stephan Schürer, Feng Chen, Maciej Wiznerowicz, Karen A Ketchum, Emily S Boja, Christopher R Kinsinger, Ana I Robles, Tara Hiltke, Mathangi Thiagarajan, Alexey I Nesvizhskii, Bing Zhang, D R Mani, Michele Ceccarelli, Xi S Chen, Sandra L Cottingham, Qing Kay Li, Albert H Kim, David Fenyö, Kelly V Ruggles, Henry Rodriguez, Mehdi Mesri, Samuel H Payne, Adam C Resnick, Pei Wang, Richard D Smith, Antonio Iavarone, Milan G Chheda, Jill S Barnholtz-Sloan, Karin D Rodland, Tao Liu, Li Ding, Clinical Proteomic Tumor Analysis Consortium Apr 2021

Proteogenomic And Metabolomic Characterization Of Human Glioblastoma, Liang-Bo Wang, Alla Karpova, Marina A Gritsenko, Jennifer E Kyle, Song Cao, Yize Li, Dmitry Rykunov, Antonio Colaprico, Joseph H Rothstein, Runyu Hong, Vasileios Stathias, Macintosh Cornwell, Francesca Petralia, Yige Wu, Boris Reva, Karsten Krug, Pietro Pugliese, Emily Kawaler, Lindsey K Olsen, Wen-Wei Liang, Xiaoyu Song, Yongchao Dou, Michael C Wendl, Wagma Caravan, Wenke Liu, Daniel Cui Zhou, Jiayi Ji, Chia-Feng Tsai, Vladislav A Petyuk, Jamie Moon, Weiping Ma, Rosalie K Chu, Karl K Weitz, Ronald J Moore, Matthew E Monroe, Rui Zhao, Xiaolu Yang, Seungyeul Yoo, Azra Krek, Alexis Demopoulos, Houxiang Zhu, Matthew A Wyczalkowski, Joshua F Mcmichael, Brittany L Henderson, Caleb M Lindgren, Hannah Boekweg, Shuangjia Lu, Jessika Baral, Lijun Yao, Kelly G Stratton, Lisa M Bramer, Erika Zink, Sneha P Couvillion, Kent J Bloodsworth, Shankha Satpathy, Weiva Sieh, Simina M Boca, Stephan Schürer, Feng Chen, Maciej Wiznerowicz, Karen A Ketchum, Emily S Boja, Christopher R Kinsinger, Ana I Robles, Tara Hiltke, Mathangi Thiagarajan, Alexey I Nesvizhskii, Bing Zhang, D R Mani, Michele Ceccarelli, Xi S Chen, Sandra L Cottingham, Qing Kay Li, Albert H Kim, David Fenyö, Kelly V Ruggles, Henry Rodriguez, Mehdi Mesri, Samuel H Payne, Adam C Resnick, Pei Wang, Richard D Smith, Antonio Iavarone, Milan G Chheda, Jill S Barnholtz-Sloan, Karin D Rodland, Tao Liu, Li Ding, Clinical Proteomic Tumor Analysis Consortium

Faculty, Staff and Students Publications

Glioblastoma (GBM) is the most aggressive nervous system cancer. Understanding its molecular pathogenesis is crucial to improving diagnosis and treatment. Integrated analysis of genomic, proteomic, post-translational modification and metabolomic data on 99 treatment-naive GBMs provides insights to GBM biology. We identify key phosphorylation events (e.g., phosphorylated PTPN11 and PLCG1) as potential switches mediating oncogenic pathway activation, as well as potential targets for EGFR-, TP53-, and RB1-altered tumors. Immune subtypes with distinct immune cell types are discovered using bulk omics methodologies, validated by snRNA-seq, and correlated with specific expression and histone acetylation patterns. Histone H2B acetylation in classical-like and immune-low GBM …


Dosing Recommendations For Vancomycin In Children And Adolescents With Varying Levels Of Obesity And Renal Dysfunction: A Population Pharmacokinetic Study In 1892 Children Aged 1–18 Years, Cornelis Smit, Sebastiaan C. Goulooze, Roger J. M. Brüggemann, Catherine M. Sherwin, Catherijne A. J. Knibbe Apr 2021

Dosing Recommendations For Vancomycin In Children And Adolescents With Varying Levels Of Obesity And Renal Dysfunction: A Population Pharmacokinetic Study In 1892 Children Aged 1–18 Years, Cornelis Smit, Sebastiaan C. Goulooze, Roger J. M. Brüggemann, Catherine M. Sherwin, Catherijne A. J. Knibbe

Pediatrics Faculty Publications

Vancomycin is an effective but potentially nephrotoxic antibiotic commonly used for severe infections. Dosing guidelines for vancomycin in obese children and adolescents with or without renal impairment are currently lacking. This study describes the pharmacokinetics of vancomycin in a large pediatric cohort with varying degrees of obesity and renal function to design practical dosing guidelines for this population. A multi-center retrospective population pharmacokinetic study was conducted using data from patients aged 1−18 years who received >1 dose of vancomycin and had ≥1 vancomycin concentration measured between January 2006 and December 2012. Besides pharmacokinetic data, age, gender, body weight, creatinine clearance …


Treatment Of Postpartum Depression With Updated Pharmacotherapy, Jennifer Yoon, Jason Gu Md, Katherine B. Martin Md Apr 2021

Treatment Of Postpartum Depression With Updated Pharmacotherapy, Jennifer Yoon, Jason Gu Md, Katherine B. Martin Md

Department of Psychiatry

No abstract provided.


Safety And Improved Efficacy Signals Following Gene Therapy In Childhood Blindness Caused By Gucy2d Mutations, Samuel G. Jacobson, Artur V. Cideciyan, Allen Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun Krishnan, Rebecca Sheplock, Sanford Boye, Alexander Dizhoor, Shannon Boye Apr 2021

Safety And Improved Efficacy Signals Following Gene Therapy In Childhood Blindness Caused By Gucy2d Mutations, Samuel G. Jacobson, Artur V. Cideciyan, Allen Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun Krishnan, Rebecca Sheplock, Sanford Boye, Alexander Dizhoor, Shannon Boye

Wills Eye Hospital Papers

A first-in-human clinical trial of gene therapy in Leber congenital amaurosis due to mutations in the GUCY2D gene is underway, and early results are summarized. A recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying the human GUCY2D gene was delivered by subretinal injection to one eye in three adult patients with severe visual loss, nystagmus, but preserved retinal structure. Safety and efficacy parameters were monitored for 9 months post-operatively. No systemic toxicity was detected; there were no serious adverse events, and ocular adverse events resolved. P1 and P2 showed statistically significant rod photoreceptor vision improvement by full-field stimulus testing in …