Medicine and Health Sciences Commons^™

Inflammatory Myopathies: Update On Diagnosis, Pathogenesis And Therapies, And Covid-19-Related Implications., Marinos C. Dalakas

Department of Neurology Faculty Papers

The inflammatory myopathies constitute a heterogeneous group of acquired myopathies that have in common the presence of endomysial inflammation. Based on steadily evolved clinical, histological and immunopathological features and some autoantibody associations, these disorders can now be classified in five characteristic subsets: Dermatomyositis (DM) Polymyositis (PM), Necrotizing Autoimmune Myositis (NAM), Anti-synthetase syndrome-overlap myositis (Anti-SS-OM), and Inclusion-Body-Myositis (IBM). Each inflammatory myopathy subset has distinct immunopathogenesis, prognosis and response to immunotherapies, necessitating the need to correctly identify each subtype from the outset to avoid disease mimics and proceed to early therapy initiation. The review presents the main clinicopathologic characteristics of each subset …

Time Cells In The Human Hippocampus And Entorhinal Cortex Support Episodic Memory., Gray Umbach, Pranish Kantak, Joshua Jacobs, Michael Kahana, Brad E. Pfeiffer, Michael R Sperling, Bradley Lega

Department of Neurology Faculty Papers

The organization of temporal information is critical for the encoding and retrieval of episodic memories. In the rodent hippocampus and entorhinal cortex, evidence accumulated over the last decade suggests that populations of "time cells" in the hippocampus encode temporal information. We identify time cells in humans using intracranial microelectrode recordings obtained from 27 human epilepsy patients who performed an episodic memory task. We show that time cell activity predicts the temporal organization of retrieved memory items. We also uncover evidence of ramping cell activity in humans, which represents a complementary type of temporal information. These findings establish a cellular mechanism …

Management Of Acute Ischemic Stroke., Franziska Herpich, Fred Rincon

Department of Neurology Faculty Papers

OBJECTIVES: Concise "synthetic" review of the state of the art of management of acute ischemic stroke.

DATA SOURCES: Available literature on PubMed.

STUDY SELECTION: We selected landmark studies, recent clinical trials, observational studies, and professional guidelines on the management of stroke including the last 10 years.

DATA EXTRACTION: Eligible studies were identified and results leading to guideline recommendations were summarized.

DATA SYNTHESIS: Stroke mortality has been declining over the past 6 decades, and as a result, stroke has fallen from the second to the fifth leading cause of death in the United States. This trend may follow recent advances in …

Contribution Of Left Supramarginal And Angular Gyri To Episodic Memory Encoding: An Intracranial Eeg Study., Daniel Y. Rubinstein, Liliana Camarillo-Rodriguez, Mijail D. Serruya, Nora A. Herweg, Zachary J. Waldman, Paul A. Wanda, Ashwini D. Sharan, Shennan A. Weiss, Michael R. Sperling

Department of Neurology Faculty Papers

The role of the left ventral lateral parietal cortex (VPC) in episodic memory is hypothesized to include bottom-up attentional orienting to recalled items, according to the dual-attention model (Cabeza et al., 2008). However, its role in memory encoding could be further clarified, with studies showing both positive and negative subsequent memory effects (SMEs). Furthermore, few studies have compared the relative contributions of sub-regions in this functionally heterogeneous area, specifically the anterior VPC (supramarginal gyrus/BA40) and the posterior VPC (angular gyrus/BA39), on a within-subject basis. To elucidate the role of the VPC in episodic encoding, we compared SMEs in the intracranial …

Challenges And Opportunities For Improving The Landscape For Lewy Body Dementia Clinical Trials., Jennifer G Goldman, Leah K Forsberg, Bradley F Boeve, Melissa J Armstrong, David J Irwin, Tanis J Ferman, Doug Galasko, James E Galvin, Daniel Kaufer, James Leverenz, Carol F Lippa, Karen Marder, Victor Abler, Kevin Biglan, Michael Irizarry, Bill Keller, Leanne Munsie, Masaki Nakagawa, Angela Taylor, Todd Graham

Department of Neurology Faculty Papers

Lewy body dementia (LBD), including dementia with Lewy bodies and Parkinson's disease dementia, affects over a million people in the USA and has a substantial impact on patients, caregivers, and society. Symptomatic treatments for LBD, which can include cognitive, neuropsychiatric, autonomic, sleep, and motor features, are limited with only two drugs (cholinesterase inhibitors) currently approved by regulatory agencies for dementia in LBD. Clinical trials represent a top research priority, but there are many challenges in the development and implementation of trials in LBD. To address these issues and advance the field of clinical trials in the LBDs, the Lewy Body …

Thalamus And Focal To Bilateral Seizures: A Multiscale Cognitive Imaging Study., Lorenzo Caciagli, Luke A Allen, Xiaosong He, Karin Trimmel, Sjoerd B Vos, Maria Centeno, Marian Galovic, Meneka K Sidhu, Pamela J Thompson, Danielle S Bassett, Gavin P Winston, John S Duncan, Matthias J Koepp, Michael R Sperling

Department of Neurology Faculty Papers

OBJECTIVE: To investigate the functional correlates of recurrent secondarily generalized seizures in temporal lobe epilepsy (TLE) using task-based fMRI as a framework to test for epilepsy-specific network rearrangements. Because the thalamus modulates propagation of temporal lobe onset seizures and promotes cortical synchronization during cognition, we hypothesized that occurrence of secondarily generalized seizures, i.e., focal to bilateral tonic-clonic seizures (FBTCS), would relate to thalamic dysfunction, altered connectivity, and whole-brain network centrality.

METHODS: FBTCS occur in a third of patients with TLE and are a major determinant of disease severity. In this cross-sectional study, we analyzed 113 patients with drug-resistant TLE (55 …

Lgi1 Encephalitis With Squamous Lung-Cell Carcinoma: Resolution After Tumor Resection., Akash Virupakshaiah, Marinos Dalakas, Neeja Desai, Scott Mintzer, Jeffrey Ratliff

Department of Neurology Faculty Papers

Encephalitis with leucine-rich glioma-inactivated-1 (LGI1) immunoglobulin G (IgG) anti-bodies classically presents with cognitive impairment and characteristic faciobrachial dystonicseizures.1In a murine model, human LGI1 IgG caused reduction of Kv1.1 channels andα-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid receptor receptors resulting in neuro-nal hyperexcitability indicating probable pathogenicity of LGI1 antibodies.2LGI1 autoimmu-nity is associated with malignancy in less than 10% of cases, including small cell lung cancer,prostate and colon cancer, squamous cell skin carcinoma, and neuroendocrine pancreaticcancer.3,4We present a case of LGI1 encephalitis only partially responsive to immunotherapywith eventual complete resolution after resection of a squamous cell lung carcinoma.

Eptinezumab For The Prevention Of Chronic Migraine: Efficacy And Safety Through 24 Weeks Of Treatment In The Phase 3 Promise-2 (Prevention Of Migraine Via Intravenous Ald403 Safety And Efficacy-2) Study., Stephen Silberstein, Merle Diamond, Nada A. Hindiyeh, David M. Biondi, Roger Cady, Joe Hirman, Brent Allan, Susan Pederson, Barbara Schaeffler, Jeff Smith

Department of Neurology Faculty Papers

BACKGROUND: PROMISE-2 was a phase 3, randomized, double-blind, placebo-controlled study that evaluated the efficacy and safety of repeat intravenous (IV) doses of the calcitonin gene-related peptide-targeted monoclonal antibody eptinezumab (ALD403) for migraine prevention in adults with chronic migraine. This report describes the results of PROMISE-2 through 24 weeks of treatment.

METHODS: Patients received up to two 30-min IV administrations of eptinezumab 100 mg, 300 mg, or placebo separated by 12 weeks. Patients recorded migraine and headache endpoints in a daily eDiary. Additional assessments, including patient-reported outcomes, were performed at regularly scheduled clinic visits throughout the 32-week study period (screening, day …

Absence-To-Bilateral-Tonic-Clonic Seizure: A Generalized Seizure Type., Sándor Beniczky, Guido Rubboli, Athanasios Covanis, Michael R Sperling

Department of Neurology Faculty Papers

OBJECTIVE: To test the hypothesis that absence seizures can evolve to generalized tonic-clonic seizures, we documented electroclinical features of this novel seizure type.

METHODS: In 4 large video-EEG databases, we identified recordings of seizures starting with impaired awareness that, without returning to baseline interictal state, evolved to generalized tonic-clonic seizures. We extracted the detailed semiologic and electrographic characteristics of these seizures, and we documented the clinical background, diagnoses, and therapeutic responses in these patients.

RESULTS: We identified 12 seizures from 12 patients. All seizures started with a period of impaired awareness and bursts of generalized spike or polyspike and slow-wave …

Progress In The Therapy Of Myasthenia Gravis: Getting Closer To Effective Targeted Immunotherapies, Marinos Dalakas

Department of Neurology Faculty Papers

PURPOSE OF REVIEW: To provide an update on immunomodulating and immunosuppressive therapies in myasthenia gravis and highlight newly approved, or pending approval, therapies with new biologics.

RECENT FINDINGS: Preoperative IVIg is not needed to prevent myasthenic crisis in stable myasthenia gravis patients scheduled for surgery under general anesthesia, based on controlled data. Rituximab, if initiated early in new-onset myasthenia gravis, can lead to faster and more sustained remission even without immunotherapies in 35% of patients at 2 years. Biomarkers determining the timing for follow-up infusions in Rituximab-responding AChR-positive patients are discussed. Most patients with MuSK-positive myasthenia gravis treated with Rituximab …

Unexpected Brain Imaging Findings In Patients With Seizures, Boulenouar Mesraoua, Matthias Koepp, Bernhard Schuknecht, Dirk Deleu, Hassan J Al Hail, Gayane Melikyan, Lubna Elsheikh, A A Asadi-Pooya

Department of Neurology Faculty Papers

New imaging technologies have advanced our ability to localize the epileptogenic zone in patients with epilepsy. As a result of the constant improvement of the image quality, magnetic resonance imaging (MRI) has become the most important ancillary tool in the management of patients with epilepsy. Magnetic resonance imaging for the evaluation of patients with epilepsy should be done using a special temporal lobe protocol and read by physicians experienced with the findings in patients with epilepsy. On the other hand, in the healthy populations, incidental structural brain abnormalities have been reported in 18% of people. Incidental, subtle, or unexpected structural …

Matrine Inhibits Cns Autoimmunity Through An Ifn-Β-Dependent Mechanism, Yaojuan Chu, Wendi Ma, Rodolfo Thome, Jie Dan Ping, Fangzhou Liu, Mengru Wang, Mingliang Zhang, Guang-Xian Zhang, Lin Zhu

Department of Neurology Faculty Papers

Matrine (MAT), a quinolizidine alkaloid component derived from the root of Sophora flavescens, suppresses experimental autoimmune encephalomyelitis (EAE), the animal model of multiple sclerosis (MS), by inducing the production of immunomodulatory molecules, e.g., IL-10. In an effort to find the upstream pathway(s) of the mechanism underlying these effects, we have tested certain upregulated immunomodulatory molecules. Among them, we found increased levels of IL-27 and IFN-β, one of the first-line MS therapies. Indeed, while low levels of IFN-β production in sera and type I interferon receptor (IFNAR1) expression in spinal cord of saline-treated control EAE mice were detected, they were significantly …

The Impact Of Fremanezumab On Medication Overuse In Patients With Chronic Migraine: Subgroup Analysis Of The Halo Cm Study., Stephen D. Silberstein, Joshua M. Cohen, Michael J. Seminerio, Ronghua Yang, Sait Ashina, Zaza Katsarava

Department of Neurology Faculty Papers

BACKGROUND: We evaluated the efficacy of fremanezumab, a fully humanized monoclonal antibody that selectively targets calcitonin gene-related peptide, in patients with chronic migraine (CM) with and without medication overuse (MO).

METHODS: In a 12-week, phase 3 trial, patients with CM were randomized to fremanezumab quarterly (675 mg/placebo/placebo), monthly (675 mg/225 mg/225 mg), or placebo. Post hoc analyses assessed the impact of fremanezumab in patients with and without MO (monthly use of acute headache medication ≥15 days, migraine-specific acute medication ≥10 days, or combination medication ≥10 days) on efficacy outcomes, including headache days of at least moderate severity (HDs), and six-item …

Dramatic Outcomes In Epilepsy: Depression, Suicide, Injuries, And Mortality, Boulenouar Mesraoua, Dirk Deleu, Al Hail Hassan, Melykian Gayane, Alsheikh Lubna, Musab Abdalhalim Ali, Torbjorn Tomson, Bassel Abou Khalil, J Helen Cross, Ali A Asadi-Pooya

Department of Neurology Faculty Papers

In this narrative review, we will discuss some of the significant risks and dramatic consequences that are associated with epilepsy: depression, suicide, seizure-related injuries, and mortality, both in adults and in children. Considering the high prevalence of depression among people with epilepsy (PWE), routine and periodic screening of all PWE for early detection and appropriate management of depression is recommended. PWE should be screened for suicidal ideation regularly and when needed, patients should be referred for a psychiatric evaluation and treatment. When starting an antiepileptic drug (AED) or switching from one to another AED, patients should be advised to report …

The Effects Of Direct Brain Stimulation In Humans Depend On Frequency, Amplitude, And White-Matter Proximity, Uma R Mohan, Andrew J Watrous, Jonathan F Miller, Bradley C Lega, Michael R Sperling, Gregory A Worrell, Robert E Gross, Kareem A Zaghloul, Barbara C Jobst, Kathryn A Davis, Sameer A Sheth, Joel M Stein, Sandhitsu R Das, Richard Gorniak, Md, Paul A Wanda, Daniel S Rizzuto, Michael J Kahana, Joshua Jacobs

Department of Neurology Faculty Papers

BACKGROUND: Researchers have used direct electrical brain stimulation to treat a range of neurological and psychiatric disorders. However, for brain stimulation to be maximally effective, clinicians and researchers should optimize stimulation parameters according to desired outcomes.

OBJECTIVE: The goal of our large-scale study was to comprehensively evaluate the effects of stimulation at different parameters and locations on neuronal activity across the human brain.

METHODS: To examine how different kinds of stimulation affect human brain activity, we compared the changes in neuronal activity that resulted from stimulation at a range of frequencies, amplitudes, and locations with direct human brain recordings. We …

Drug-Resistant Epilepsy In Children With Juvenile Huntington's Disease: A Challenging Case And Brief Review, Abdulhafeez Khair, Jessica Kabrt, Stephen Falchek

Department of Neurology Faculty Papers

Huntington's Disease (HD) is an autosomal dominant neurodegenerative disorder with a progressive decline in cognitive, motor, and psychological function. Chorea tends to be the most common associated movement disorder, although other variants of several abnormal movements are also seen. Adult-onset HD is the most common subtype. Juvenile Huntington's disease (JHD) accounts for 5%-10% of all HD cases and presents as a rapidly progressive disorder with a multitude of characteristics. We report on a 9-year-old male with JHD who presented with refractory epilepsy. His EEG findings, seizure type, and antiepileptic drug usage are discussed with a brief review of the currently …

Dimethyl Fumarate Suppresses Granulocyte Macrophage Colony-Stimulating Factor-Producing Th1 Cells In Cns Neuroinflammation., Farinaz Safavi, Rodolfo Thome, Zichen Li, Guang-Xian Zhang, Abdolmohamad Rostami

Department of Neurology Faculty Papers

Objective To study the immunomodulatory effect of dimethyl fumarate (DF) on granulocyte macrophage colony-stimulating factor (GM-CSF) production in CD4+ T cells in experimental autoimmune encephalomyelitis (EAE) and human peripheral blood mononuclear cells (PBMCs).

Methods We collected splenocytes and CD4+ T cells from C57BL/6 wild-type and interferon (IFN)-γ–deficient mice. For human PBMCs, venous blood was collected from healthy donors, and PBMCs were collected using the Percoll gradient method. Cells were cultured with anti-CD3/28 in the presence/absence of DF for 3 to 5 days. Cells were stained and analyzed by flow cytometry. Cytokines were measured by ELISA in cell supernatants. For in …

Innervation: The Missing Link For Biofabricated Tissues And Organs, Suradip Das, Wisberty J. Gordián-Vélez, Harry C. Ledebur, Foteini Mourkiot, Panteleimon Rompolas, H. . Isaac Chen, Mijail D. Serruya, D. Kacy Cullen

Department of Neurology Faculty Papers

Innervation plays a pivotal role as a driver of tissue and organ development as well as a means for their functional control and modulation. Therefore, innervation should be carefully considered throughout the process of biofabrication of engineered tissues and organs. Unfortunately, innervation has generally been overlooked in most non-neural tissue engineering applications, in part due to the intrinsic complexity of building organs containing heterogeneous native cell types and structures. To achieve proper innervation of engineered tissues and organs, specific host axon populations typically need to be precisely driven to appropriate location(s) within the construct, often over long distances. As such, …

Posterior Cortical Atrophy: Characteristics From A Clinical Data Registry., Jennifer J Olds, William L Hills, Judith Warner, Julie Falardeau, Lori Haase Alasantro, Mark L Moster, Robert A Egan, Wayne T Cornblath, Andrew G Lee, Benjamin M Frishberg, Roger E Turbin, David M Katz, John A Charley, Victoria S Pelak

Department of Neurology Faculty Papers

No abstract provided.

Zinc Finger Rna-Binding Protein Zn72d Regulates Adar-Mediated Rna Editing In Neurons., Anne L Sapiro, Emily C Freund, Lucas Restrepo, Huan-Huan Qiao, Amruta Bhate, Qin Li, Jian-Quan Ni, Timothy J Mosca, Jin Billy Li

Department of Neurology Faculty Papers

Adenosine-to-inosine RNA editing, catalyzed by adenosine deaminase acting on RNA (ADAR) enzymes, alters RNA sequences from those encoded by DNA. These editing events are dynamically regulated, but few trans regulators of ADARs are known in vivo. Here, we screen RNA-binding proteins for roles in editing regulation with knockdown experiments in the Drosophila brain. We identify zinc-finger protein at 72D (Zn72D) as a regulator of editing levels at a majority of editing sites in the brain. Zn72D both regulates ADAR protein levels and interacts with ADAR in an RNA-dependent fashion, and similar to ADAR, Zn72D is necessary to maintain proper neuromuscular …

Nk Cell-Derived Gm-Csf Potentiates Inflammatory Arthritis And Is Negatively Regulated By Cis, Cynthia Louis, Fernando Souza-Fonseca-Guimaraes, Yuyan Yang, Damian D'Silva, Tobias Kratina, Laura Dagley, Soroor Hediyeh-Zadeh, Jai Rautela, Seth Lucian Masters, Melissa J Davis, Jeffrey J Babon, Bogoljub Ciric, Eric Vivier, Warren S Alexander, Nicholas D Huntington, Ian P Wicks

Department of Neurology Faculty Papers

Despite increasing recognition of the importance of GM-CSF in autoimmune disease, it remains unclear how GM-CSF is regulated at sites of tissue inflammation. Using GM-CSF fate reporter mice, we show that synovial NK cells produce GM-CSF in autoantibody-mediated inflammatory arthritis. Synovial NK cells promote a neutrophilic inflammatory cell infiltrate, and persistent arthritis, via GM-CSF production, as deletion of NK cells, or specific ablation of GM-CSF production in NK cells, abrogated disease. Synovial NK cell production of GM-CSF is IL-18–dependent. Furthermore, we show that cytokine-inducible SH2-containing protein (CIS) is crucial in limiting GM-CSF signaling not only during inflammatory arthritis but also …

The Lysosomal Diseases Testing Laboratory: A Review Of The Past 47 Years., David A. Wenger, Paola Luzi

Department of Neurology Faculty Papers

Lysosomal disorders are diseases that involve mutations in genes responsible for the coding of lysosomal enzymes, transport proteins, activator proteins and protein processing enzymes. These defects lead to the storage of specific metabolites within lysosomes resulting in a great variety of clinical features depending on the tissues with the storage, the storage products and the extent of the storage. The methods for rapidly diagnosing patients started in the late 1960's when the enzyme defects were identified eliminating the need for tissue biopsies. The first requests for diagnostic help in this laboratory came in 1973. In that year, patients with Krabbe …

Spider Venom Administration Impairs Glioblastoma Growth And Modulates Immune Response In A Non-Clinical Model., Amanda Pires Bonfanti, Natália Barreto, Jaqueline Munhoz, Marcus Caballero, Gabriel Cordeiro, Thomaz Rocha-E-Silva, Rafael Sutti, Fernanda Moura, Sérgio Brunetto, Celso Dario Ramos, Rodolfo Thome, Liana Verinaud, Catarina Rapôso

Department of Neurology Faculty Papers

Molecules from animal venoms are promising candidates for the development of new drugs. Previous in vitro studies have shown that the venom of the spider Phoneutria nigriventer (PnV) is a potential source of antineoplastic components with activity in glioblastoma (GB) cell lines. In the present work, the effects of PnV on tumor development were established in vivo using a xenogeneic model. Human GB (NG97, the most responsive line in the previous study) cells were inoculated (s.c.) on the back of RAG-/- mice. PnV (100 µg/Kg) was administrated every 48 h (i.p.) for 14 days and several endpoints were evaluated: tumor …

Bone Marrow Dendritic Cells Deficient For Cd40 And Il-23p19 Are Tolerogenic In Vitro, Tahereh Kalantari, Bogoljub Ciric, Eskandar Kamali-Sarvestani, Abdolmohamad Rostami

Department of Neurology Faculty Papers

Objective(s): In addition to pro-inflammatory role, dendritic cells (DCs) can also be anti-inflammatory when they acquire tolerogenic phenotype. In this study we tested the role of CD40 and IL-23p19 in antigen presenting function of bone marrow-derived DCs (BMDCs) by comparing BMDCs derived from CD40 knockout (CD40KO-DCs) and IL-23p19 (IL-23p19KO-DCs) knockout mice with those from C57BL/6 mice (Cont-DCs). We have focused on CD40 and IL-23, as these molecules have well established pro-inflammatory roles in a number of autoimmune and inflammatory diseases.

Materials and Methods: The expression of maturation markers MHC II and co-stimulatory molecules CD40, CD80, and CD86 was analyzed by …

Evaluation Of The Effect Of Gm-Csf Blocking On The Phenotype And Function Of Human Monocytes., Noushin Lotfi, Guang-Xian Zhang, Nafiseh Esmaeil, Mohamad Rostami

Department of Neurology Faculty Papers

Granulocyte-macrophage colony-stimulating factor (GM-CSF) is a multipotent cytokine that prompts the proliferation of bone marrow-derived macrophages and granulocytes. In addition to its effects as a growth factor, GM-CSF plays an important role in chronic inflammatory autoimmune diseases such as multiple sclerosis and rheumatoid arthritis. Reports have identified monocytes as the primary target of GM-CSF; however, its effect on monocyte activation has been under-estimated. Here, using flow cytometry and ELISA we show that GM-CSF induces an inflammatory profile in human monocytes, which includes an upregulated expression of HLA-DR and CD86 molecules and increased production of TNF-α and IL-1β. Conversely, blockage of …

Evaluation Of The Effect Of Gm-Csf Blocking On The Phenotype And Function Of Human Monocytes, Noushin Lotfi, Guang-Xian Zhang, Nafiseh Esmaeil, Mohamad Rostami

Department of Neurology Faculty Papers

Granulocyte-macrophage colony-stimulating factor (GM-CSF) is a multipotent cytokine that prompts the proliferation of bone marrow-derived macrophages and granulocytes. In addition to its effects as a growth factor, GM-CSF plays an important role in chronic inflammatory autoimmune diseases such as multiple sclerosis and rheumatoid arthritis. Reports have identified monocytes as the primary target of GM-CSF; however, its effect on monocyte activation has been under-estimated. Here, using flow cytometry and ELISA we show that GM-CSF induces an inflammatory profile in human monocytes, which includes an upregulated expression of HLA-DR and CD86 molecules and increased production of TNF-α and IL-1β. Conversely, blockage of …

Potential Roles Of Extracellular Vesicles In The Pathophysiology, Diagnosis, And Treatment Of Autoimmune Diseases., Jing Tian, Giacomo Casella, Yuan Zhang, Abdolmohamad Rostami, Xing Li

Department of Neurology Faculty Papers

Since extracellular vesicles (EVs) were discovered in 1983 in sheep reticulocytes samples, they have gradually attracted scientific attention and become a topic of great interest in the life sciences field. EVs are small membrane particles, released by virtually every cell that carries a variety of functional molecules. Their main function is to deliver messages to the surrounding area in both physiological and pathological conditions. Initially, they were thought to be either cell debris, signs of cell death, or unspecific structures. However, accumulating evidence support a theory that EVs are a universal mechanism of communication. Thanks to their biological characteristics and …

Conditions For Combining Gene Therapy With Bone Marrow Transplantation In Murine Krabbe Disease., Mohammad Rafi, Paola Luzi, David A Wenger

Department of Neurology Faculty Papers

No abstract provided.

Emerging Regenerative Medicine And Tissue Engineering Strategies For Parkinson's Disease., James P Harris, Justin C Burrell, Laura A Struzyna, H Isaac Chen, Mijail D Serruya, John A Wolf, John E Duda, D Kacy Cullen

Department of Neurology Faculty Papers

Parkinson's disease (PD) is the second most common progressive neurodegenerative disease, affecting 1-2% of people over 65. The classic motor symptoms of PD result from selective degeneration of dopaminergic neurons in the substantia nigra pars compacta (SNpc), resulting in a loss of their long axonal projections to the striatum. Current treatment strategies such as dopamine replacement and deep brain stimulation (DBS) can only minimize the symptoms of nigrostriatal degeneration, not directly replace the lost pathway. Regenerative medicine-based solutions are being aggressively pursued with the goal of restoring dopamine levels in the striatum, with several emerging techniques attempting to reconstruct the …