861 results on '"Byrne, Barry J."'
Search Results
202. Airway smooth muscle dysfunction in Pompe (Gaa-/-) mice.
203. Mutation-adapted U1 snRNA corrects a splicing error of the dopa decarboxylase gene.
204. Intramyocardial injection of autologous bone marrow mononuclear cells for patients with chronic ischemic heart disease and left ventricular dysfunction ( First M ononuclear Cells injected in the US [FOCUS]): Rationale and design
205. Cell Therapy in the Heart: Cell Production, Transplantation, and Applications
206. Gene Therapy for Cardiovascular Disease
207. Peripheral nerve and neuromuscular junction pathology in Pompe disease
208. Magnetic Resonance Imaging and Spectroscopy Assessment of Lower Extremity Skeletal Muscles in Boys with Duchenne Muscular Dystrophy: A Multicenter Cross Sectional Study
209. Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients with Pompe Disease
210. Charting a Clear Path: The ASGCT Standardized Pathways Conference
211. New Mechanistic and Therapeutic Targets for Pediatric Heart Failure
212. Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent
213. Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice
214. Treatment of Congenital Neurotransmitter Deficiencies by Intracerebral Ventricular Injection of an Adeno-Associated Virus Serotype 9 Vector
215. Strategy to assess the effect of duvoglustat co-administered with alglucosidase alfa infusion on the immune response to enzyme replacement therapy for Pompe disease
216. Pharmacological chaperones prevent the precipitation of rhGAA by anti-GAA antibodies during enzyme replacement therapy
217. A simplified purification protocol for recombinant adeno-associated virus vectors
218. Adeno-Associated Virus–Mediated Gene Therapy for Metabolic Myopathy
219. The respiratory neuromuscular system in Pompe disease
220. Skeletal Muscles of Ambulant Children with Duchenne Muscular Dystrophy: Validation of Multicenter Study of Evaluation with MR Imaging and MR Spectroscopy
221. Intracardiac foreign body in a dog
222. Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease
223. Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 Human Acid α-Glucosidase
224. Pathway for Approval of a Gene Therapy Orphan Product: Treading New Ground
225. Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes
226. Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012
227. Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
228. Magnetic resonance imaging and spectroscopy to evaluate muscle involvement in Pompe disease
229. Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice
230. Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.
231. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
232. Respiratory motor function in individuals with centronuclear myopathies.
233. Tadalafil Alleviates Muscle Ischemia in Patients with Becker Muscular Dystrophy
234. A degradable, bioactive, gelatinized alginate hydrogel to improve stem cell/growth factor delivery and facilitate healing after myocardial infarction
235. The Barth Syndrome Registry: Distinguishing disease characteristics and growth data from a longitudinal study
236. MRI/MRS evaluation of a female carrier of Duchenne muscular dystrophy
237. Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment
238. An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery
239. Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9
240. Mapping the T helper cell response to acid α-glucosidase in Pompe mice
241. Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy
242. Gene Therapy for Aromatic l -Amino Acid Decarboxylase Deficiency
243. Substrate metabolism during basal and hyperinsulinemic conditions in adolescents and young‐adults with Barth syndrome
244. Chronic Intramuscular Electrodes To Evaluate Diaphragm Inspiratory Load Compensation In A Ventilated Adult
245. Retrograde Gene Delivery to Hypoglossal Motoneurons Using Adeno-Associated Virus Serotype 9
246. AN INJECTABLE ACELLULAR CAPILLARY HYDROGEL IMPROVES LEFT VENTRICULAR FUNCTION AFTER MYOCARDIAL INFARCTION
247. Signs of Progress in Gene Therapy for Muscular Dystrophy Also Warrant Caution
248. A New Approach to Winning: Insulin-Like Growth Factor-I Signaling in Competitive Sport
249. Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice
250. Combination therapy for inhibitor reversal in haemophilia A using monoclonal anti-CD20 and rapamycin
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.