About

Brandon Jones is an Assistant Professor of Clinical Neurology in the Department of Neurology at the Perelman School of Medicine, University of Pennsylvania. He completed his BS in Microbiology, MS in Neuroscience, and MD at Louisiana State University Health, New Orleans School of Medicine, in 2011, 2013, and 2019 respectively. His research and clinical interests focus on neurology, with particular attention to epilepsy, sleep disorders, and migraine treatment. Dr. Jones has contributed to advancing understanding in these areas through various research projects, including studies on the risk of ADHD in children with childhood absence epilepsy, predicting responses to migraine preventive medications using machine learning models, and exploring sleep behavior disorders such as REM sleep behavior disorder. His work involves applying innovative methodologies to improve diagnosis and treatment strategies in neurology.

Research topics

• Chromatography
• Molecular biology
• Internal medicine
• Biochemistry
• Medicine
• Chemistry
• Biology

Selected publications

• Loss of factor VIII in zebrafish rebalances antithrombin deficiency but has a limited bleeding diathesis

  Blood Advances · 2025-03-14 · 2 citations

  articleOpen access

  ABSTRACT: Deficiencies in coagulation factor VIII (FVIII, F8) result in the bleeding disorder hemophilia A. An emerging novel therapeutic strategy for bleeding disorders is to enhance hemostasis by limiting natural anticoagulants, such as antithrombin (AT3). To study pro/anticoagulant hemostatic balance in an in vivo model, we used genome editing to create null alleles for f8 and von Willebrand factor (vwf) in zebrafish, a model organism with a high degree of homology to the mammalian hemostatic system and unique attributes, including external development and optical transparency. f8 homozygous mutant larvae surprisingly formed normal thrombi when subjected to laser-mediated endothelial injury, had no overt signs of hemorrhage, although they did have a modest increase in mortality. We have previously shown that at3-/- larvae develop disseminated intravascular coagulation (DIC), with spontaneous thrombosis and fibrinogen consumption, resulting in a bleeding phenotype marked by secondary lack of induced thrombus formation upon endothelial injury. We found that with loss of FVIII (f8-/-;at3-/-), larvae no longer developed spontaneous fibrin thrombi and produced clots in response to endothelial injury. In contrast, homozygous loss of zebrafish Vwf failed to rescue the at3 DIC phenotype. These findings demonstrate an altered balance of natural anticoagulants that mitigates FVIII deficiency in zebrafish, similar to hemostatic drugs in the clinical development pipeline, and suggest that zebrafish FVIII might circulate independently of Vwf. Further exploration of this unique balance in zebrafish could provide novel insights into the treatment of hemophilia A and von Willebrand disease.

  PublisherDOI

• Use of CD19-targeted immune modulation to eradicate AAV-neutralizing antibodies

  Molecular Therapy · 2025-03-08 · 6 citations

  articleOpen access

  Neutralizing antibodies (NAbs) against adeno-associated virus (AAV) represent a significant obstacle to the efficacy of systemic recombinant AAV vector administration or re-administration. While there are some promising preclinical immunomodulation strategies in development, insights into which B cell subsets and compartments maintain persistent AAV NAb may define the optimal eradication strategy. Given the limited success of CD20-directed monotherapy in previous studies, we hypothesized that CD19-directed approaches that extend targeting into the plasma cell compartments may improve AAV NAb eradication. We tested this approach in mice using chimeric antigen receptor T (CAR-T) cells or monoclonal antibodies (mAbs). We observed that combination mAbs targeting CD19, CD22, CD20, or B220 in mice did not eliminate tissue-resident B cells and, correspondingly, did not deplete pre-existing high titer AAV8 NAb. In contrast, CD19 CAR-T therapy eliminated peripheral and tissue-resident B cells and plasma cells and resulted in a marked reduction or eradication of high titer AAV8 NAb that permitted successful transgene expression following systemic AAV8 re-administration in mice. This successful therapeutic approach in mice identifies the population and location of B cells necessary to reduce or eradicate AAV NAb sufficiently to permit successful transgene expression with systemic AAV vector administration.

  PublisherOA PDFDOI

• Ancient origin of the furin sequence in the wolf F8 gene

  Open Veterinary Journal · 2025-01-01

  articleOpen access

  Background: Our previous characterization of canine coagulation factor VIII (FVIII) in the naturally occurring hemophilia A (HA) dog models provided insight into the evolution of the FVIII protein. We noted a unique sequence (H1645) in the R-X-X-R furin-recognition motif within the B domain of canine FVIII that was distinct from the sequence (R1645) in humans and other model organism species, including mice, rats, pigs, and sheep. R1645 was associated with lower secretion and biological activity, which can be improved with the canine H1645 sequence. Aim: Herein, we sought to determine the evolutionary origin of the canine H1645. Methods: ), in several free-range animals from diverse geographic locations and compared it to several breeds of domestic dogs. We also compared our sequences to publicly available reference sequences for other members of the class Mammalia, order Carnivora, and suborder Caniformia. Results: 19 X chromosomes from 12 gray wolves, at least 20 X chromosomes from 14 coyotes, and at least 12 X chromosomes from 12 domestic dogs of at least 10 distinct breeds all had the canine H1645 variant. Other members of the order Carnivora and suborder Caniformia, whose sequences are publicly available, had the R1645 sequence. Conclusion: Our results suggest that the H1645 variant in the furin-consensus sequence was likely derived after the infraorders Cynoidea and Arctoidea diverged but before the separation of the gray wolf and coyote and persists through the domestic dog.

  PublisherDOI

• Fidanacogene Elaparvovec for Hemophilia B — A Multiyear Follow-up Study

  New England Journal of Medicine · 2025-04-16 · 18 citations

  articleOpen access

  BACKGROUND: Treatment with fidanacogene elaparvovec, a recombinant adeno-associated virus (AAV) vector developed for the treatment of hemophilia B, led to sustained expression of the high-activity factor IX variant (FIX-R338L, or FIX-Padua) in a phase 1-2a study. The long-term safety and efficacy of this treatment are not known. METHODS: vector genomes (vg) per kilogram of body weight; thereafter, participants could enroll in a 5-year follow-up study. Safety end points included adverse events and changes in laboratory measures. Efficacy end points included the annualized rate of treated bleeding events (annualized bleeding rate) and factor IX activity. RESULTS: A total of 14 participants provided consent and completed at least 3 years of follow-up (median, 5.5; range 3 to 6); participation was ongoing among 8 at the data cutoff. None of the participants reported treatment-related adverse events after year 1. Throughout follow-up, nine serious adverse events were noted in 4 participants; none were thrombotic or treatment-related. No factor IX inhibitors were detected. Throughout follow-up, mean factor IX activity was in the mild hemophilia range; the mean annualized bleeding rate was less than 1, and 10 participants had no treated bleeding episodes. Surveillance liver ultrasounds obtained from year 1 onward showed no evidence of cancer but showed steatosis in 4 participants who had weight gain and elevated aminotransferase levels (maximum alanine aminotransferase level, 77 U per liter). One participant with a history of hepatitis C, hepatitis B, human immunodeficiency virus infection, and an elevated body-mass index had progression of underlying advanced liver fibrosis. A total of 13 surgical procedures were performed in 8 participants; exogenous factor IX was administered for 10 procedures, and no associated unexpected bleeding complications occurred. CONCLUSIONS: vg per kilogram, one of the lowest intravenous doses of AAV used for any indication. (Funded by Pfizer; ClinicalTrials.gov number, NCT03307980.).

  PublisherOA PDFDOI

• The timely reporting of clinical trial results answers 1 unknown for hemophilia gene therapy: no benefit of prophylactic immunosuppression

  Journal of Thrombosis and Haemostasis · 2025-04-28

  articleSenior author
  PublisherDOI

• Hypertension and encephalopathy in a pediatric patient following red blood cell transfusion

  Transfusion · 2025-04-26

  articleSenior author

  BACKGROUND: Post-transfusion encephalopathy is a rare complication of red blood cell transfusions. Diagnostic entities include reversible cerebral vasoconstriction syndrome (RCVS) and posterior reversible encephalopathy syndrome (PRES), which can be difficult to distinguish. These have primarily been reported in adults with severe chronic anemia. There are limited reports of these entities occurring in children after transfusions; most reported cases have clear pre-existing risk factors. STUDY DESIGN AND METHODS: Here we describe a 4-year-old male who presented with altered mental status 5 days after being treated for severe iron-deficiency anemia with large volume packed red blood transfusions, where his hemoglobin was increased from 1.4 to 11.4 g/dL over 70 h. Brain magnetic resonance imaging was concerning for RCVS/PRES. He made a complete neurologic recovery after 8 days with aggressive blood pressure control with calcium channel blockers. RESULTS AND DISCUSSION: Though this case had features of both RCVS and PRES, his hypertension and encephalopathy were in retrospect most consistent with PRES. This case highlights both the diagnostic challenges related to and important features of transfusion-related cerebral pathology, including large volume red blood cell transfusion for severe chronic anemia, even when administered over multiple days. Prompt recognition of transfusion-related neurologic disease is critical for appropriate management.

  PublisherDOI

• Ex vivo lentiviral gene therapy for severe hemophilia A: an alternative to recombinant adeno-associated viral-based strategies?

  Research and Practice in Thrombosis and Haemostasis · 2025-02-01

  articleOpen access1st authorCorresponding
  PublisherDOI

• Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns

  Molecular Therapy · 2025-10-05 · 3 citations

  articleOpen access
  PublisherOA PDFDOI

• HTRS2025.P1.47 Procoagulant Enhancement of Inherited Factor X Deficiency with Emicizumab

  Research and Practice in Thrombosis and Haemostasis · 2025-11-01

  articleOpen accessSenior author

  Non-adherence to DOACs was defined as PDC < 0.80.Recurrent thrombosis, stroke, major bleeding and clinically relevant non-major bleeding (CRNMB) events were collected.Results: 57 patients were started on DOACs: 53 (93%) were treated with rivaroxaban and 4 (7%) with apixaban.46 (81%) were for the indication of venous thromboembolism, 5 (9%) were for stroke, 4 (7%) were for venous malformation, and 2 (3%) were for atrial fibrillation.The median age was 14 years (range: 0.6-17), and the mean duration of treatment exposure was 6.7 months ( 4.5).The mean PDC was 0.87 (0.17); 26.3% were non-adherent patients (PDC < 0.80).Eight (14%) patients prematurely self-discontinued DOACs during their treatment period.Two of these patients experienced recurrent thrombotic events (one had recurrent stroke, the other had recurrent venous thromboembolism) and resumed their DOAC during the hospital admission.There were no major bleeding events; eight (14%) experienced CRNMB.Seven (37%) of 19 post-menarchal patients experienced heavy menstrual bleeding which accounted for 89% of the CRNMB events.Conclusions: Our ongoing real-world study suggests more than one quarter of patients had poor DOAC adherence.Premature self-discontinuation of DOAC may lead to recurrent thrombotic events.Targeted interventions to enhance DOAC adherence in children are urgently needed.Other: 1.

  PublisherOA PDFDOI

• “An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B”: comment from Lee et al.

  Journal of Thrombosis and Haemostasis · 2025-07-28 · 1 citations

  letterOpen accessSenior author
  PublisherOA PDFDOI

Frequent coauthors

• Margaret V. Ragni

  University of Pittsburgh

  107 shared

• Margareth C. Ozelo

  Universidade Estadual de Campinas (UNICAMP)

  102 shared

• Leonard A. Valentino

  Rush University Medical Center

  100 shared

• Nigel S. Key

  University of North Carolina at Chapel Hill

  100 shared

• Allyson M. Pishko

  University of Pennsylvania

  100 shared

• David Lillicrap

  Queen's University

  100 shared

• Roland W. Herzog

  Indiana University – Purdue University Indianapolis

  100 shared

• Valder R. Arruda

  65 shared

Labs

• Brandon Jones LabPI

Education

• Pediatric Hematology and Oncology Fellowship

  The Children's Hospital of Philadelphia

  2015

• Pediatrics Residency

  Children's National Medical Center

  2012

• MD/PhD

  Albert Einstein College of Medicine

  2009

• BA

  Amherst College

  2001