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Topic: Paola Leone


  
  Jefferson researchers use gene therapy for rare, inherited brain disease
Leone, Freese and Matthew During, M.D., professor of neurosurgery at Jefferson Medical College, then all at Yale University, were contacted by families with children with Canavan disease to see about the possibility of developing a therapy for the disease.
During, Leone and Freese developed a gene delivery system based on liposomes, or fatty molecules, and polymers, rather than viruses, to deliver the genetic information for the enzyme into the brain.
Several photographs taken by Dr. Leone showing patients, families, doctors and researchers participating in the earlier trial currently are on display at The Genomic Revolution exhibit at the American Museum of Natural History in New York until January 1, 2002.
www.eurekalert.org /pub_releases/2001-07/tju-jru071001.php   (686 words)

  
 The Myelin Project: June 2003 Progress Report
Paola Leone from Robert Wood Johnson Medical School in New Jersey is forging ahead with her gene therapy trial in children with Canavan disease.
You will recall that during her stay at Thomas Jefferson University, Dr. Paola Leone in 2001 had performed gene therapy in three Canavan children, under a protocol involving infusion into the brain of a liquid solution containing several billion particles of an adeno-associated virus.
Leone reports that in the 18 months following surgery, the treated children showed normalization of neurochemistry in the brain and increases in their myelin content.
www.myelin.org /06232003.htm   (3510 words)

  
 Ilyce and Max Randell - Daily Herald article
In 2001, Leone and her research team at Jefferson started using a more potent viral delivery system for the gene therapy.
Leone then operates a pump that injects the virus, carrying the aspartoacylase gene, into the children's brains.
Leone says that research is about $800,000 away from a clinical study, again emphasizing the importance of fund raising.
www.canavanresearch.org /DailyHerald.htm   (1389 words)

  
 Canavan Research Illinois - Medical Research to Treat and Cure Canavan Disease
Leone is also the Principal Investigator of the Canavan Gene Therapy protocol using adeno-associated viral vectors.
From 1998-2001, she was Associate Director of the CNS Gene Therapy Center at Jefferson Medical College and an Assistant Professor in the Department of Neurosurgery.
Leone has been responsible for the development and characterization of viral (AAV, adenovirus, retrovirus) and non-viral vectors for the treatment of Canavan Disease and other disorders.
www.canavanresearch.org /forward.htm   (503 words)

  
 Girl's Parents Plead for Gene Therapy to Resume
Paola Leone, PhD, is an associate director of the GNS Gene Therapy Center at the Thomas Jefferson University Medical College and the sponsor of Lindsay's genetic therapy.
The next phase, Leone says, calls for injecting a much higher number of these genes into Lindsay's brain, which may even halt the progression of her disease.
The FDA does not comment on pending petitions, but the Karlins say agency officials have agreed to revisit Leone's application, which they say shows that authorities are beginning to share researchers' worries about the effect Gelsinger's death may have on gene therapy.
my.webmd.com /content/article/28/1728_61753   (987 words)

  
 Cell & Gene Therapy Center - Faculty & Staff   (Site not responding. Last check: 2007-10-17)
Paola Leone, Ph.D. Dr. Paola Leone is an Associate Professor of Neurosurgery and Director of the Cell and Gene Therapy Center (CGTC).
She has published extensively in the field of CNS gene therapy and conducted the first direct gene therapy trial using a liposome-based vector for a pediatric neurodegenerative disorder.
In addition to applications of viral vectors and stem cells, Dr. Leone is studying pharmacological approaches in humans and in animal models of Canavan Disease, Amyotrophic Lateral Sclerosis, Tay Sach’s and other neurological disorders.
www4.umdnj.edu /cgtcweb/faculty   (1068 words)

  
 Endeavors, UNC-Chapel Hill : Fall 2003
Leone is principal investigator for a phase-one safety study testing whether some of Samulski's adeno-associated virus, prepackaged with flawless ASPA genes, can actually reverse the damage to Canavan brains by replacing the flawed genes.
In April, a day before Lana Swancey's second birthday, Leone's team of neurosurgeons inserted six fine catheters into the child's brain and in less than an hour carefully pumped 210 microliters of solution bearing about 900 billion genomic particles into specific areas of the brain chosen with minute care.
Leone had turned to Samulski and his team, including Angelique Camp and Scott McPhee, to produce clinical-grade AAV vector for her study.
research.unc.edu /endeavors/fall2003/canavan.html   (1339 words)

  
 SSM Online - News   (Site not responding. Last check: 2007-10-17)
National Institute of Neurological Disorders and Stroke, is under the direction of Cooper neuroscientist Paola Leone, PhD, principal investigator of the study and director, Cell and Gene Therapy Center at Cooper.
Currently, Dr Leone’s research study is in Phase I. From now until 2005, 21 Canavan patients will receive this treatment and will be evaluated, studied, and tracked for clinical progress and outcomes under the guidelines of the study.
Dr Leone and her research team anticipate that the collection and analysis of detailed safety data and associated clinical outcomes from this study will provide the basis for larger (Phase II/III) clinical trials, and that this study will be useful as a reference for all future trials using viral vectors in the human brain.
www.ssmonline.org /News/ViewRelease.asp?ReleaseID=3297   (702 words)

  
 Applied Genetics News: Canavan Trial Called Promising   (Site not responding. Last check: 2007-10-17)
Matthew During and Paula Leone, at Jefferson Medical College, who led the 1996 trial in New Zealand--a prelude to a larger study at Jefferson in 1998 and 1999 of 14 such patients-- say the report bodes well for future use of gene therapy for brain diseases.
During, Leone and their co-workers report the details of the trial in July in the Annals of Neurology.
Overall, we think it was an encouraging result for a preliminary gene transfer trial," says Paola Leone, assistant professor of neurosurgery at Jefferson Medical College and associate director of the CNS gene therapy center.
www.findarticles.com /p/articles/mi_m0DED/is_1_21/ai_65016215   (464 words)

  
 Beat Canavan Disease, and save a little girl's life   (Site not responding. Last check: 2007-10-17)
Dr. Paola Leone and her team are the only researchers in the world working directly with Canavan children.
Leone and her team have pioneered a brain gene therapy to halt the progression of the disease.
Their research offers a glimpse at treating and eventually eradicating similar degenerative diseases of the brain, such as Parkinson's disease, Alzheimer's disease, ALS (Lou Gehrig's disease), and Multiple Sclerosis.
www.beatcanavan.org   (354 words)

  
 Journal of Cerebral Blood Flow & Metabolism - Abstract of article: Striking Differences in Glucose and Lactate ...   (Site not responding. Last check: 2007-10-17)
Supported by grants from the National Institutes of Health (DK 20495, DK 45735, RR 00125, AA 00261, NS 39093) and the Juvenile Diabetes Research Foundation Center for the Study of Hypoglycemia at Yale.
Leone and During are currently at Thomas Jefferson University, Philadelphia, PA, U.S.A. Received 17 July 2001; Revised 22 October 2001; Accepted 22 October 2001.
Brain levels of glucose and lactate in the extracellular fluid (ECF), which reflects the environment to which neurons are exposed, have never been studied in humans under conditions of varying glycemia.
www.nature.com /jcbfm/journal/v22/n3/abs/9591215a.html   (341 words)

  
 ORTHO 630: Orthodontic Clinic
Paola Leone / 2 Credits / Quarters 9-11*
Direct clinical application of principles of orthodontic diagnosis and treatment planning for simple orthodontic appliances to modify tooth position in preparation for definitive restorative and/or periodontal therapy.
Final grade is Pass/Fail/Honors based on performance during lectures and clinic.
www.dental.washington.edu /sodoc/ORTHO/630.htm   (162 words)

  
 Latest Breaking Health News & Information: Applesforhealth.com
"We are blessed to have found Dr. Leone," said Michelle Swancey, Lana's mother, who learned about the NIH research study through the Internet.
Lana is one of seven patients enrolled in the research study who will undergo treatment this year.
The Swancey family met with Dr. Leone in December 2001 when Lana was six months old.
www.applesforhealth.com /OrganDonation/todbratrasu4.html   (321 words)

  
 Thomas Jefferson University Hospital - Jefferson Researchers Use Gene Therapy to Treat Rare, Inherited Brain Disease   (Site not responding. Last check: 2007-10-17)
The group includes Andrew Freese, M.D., Ph.D., associate professor of neurosurgery and director of neurosurgery research, Matthew During, M.D., professor of neurosurgery, Paola Leone, Ph.D., adjunct assistant professor of neurosurgery, and Giancarlo Barolat, M.D., professor of neurosurgery, as well as their colleagues there and at Yale University.
According to Dr. Freese, three years ago, his colleagues at Yale were contacted by Canavan families for their expertise in gene therapy to see about the possibility of developing a therapy for the disease.
During, Leone and Freese developed a gene delivery system based on liposomes and polymers, rather than viruses, to deliver the genetic information for the enzyme into the brain.
www.jeffersonhospital.org /news/1998/article7379.html   (762 words)

  
 Canavan Research Foundation
Paola Leone, Ph.D. Robert Wood Johnson Medical School, Director of the Cell and Gene Therapy Center and Associate Professor, Neurosurgery; Coriell Center for Medical Research, Adjunct Member
Leone is currently Principal Investigator on the Canavan gene therapy protocol using adeno-associated viral vectors.
Her laboratory is conducting in-vivo studies of both viral vectors and stem cells and their use in neural transplantation for therapeutic applications on a variety of neurodegenerative disorders, brain and spinal injuries and stroke.
www.canavan.org /research.htm   (576 words)

  
 CRS4 Visual Computing (ViC) / publications   (Site not responding. Last check: 2007-10-17)
Gassan Abdoulaev, Sandro Cadeddu, Giovanni Delussu, Marco Donizelli, Luca Formaggia, Andrea Giachetti, Enrico Gobbetti, Andrea Leone, Cristina Manzi, Piero Pili, Alan Scheinine, Massimiliano Tuveri, Alberto Varone, Alessandro Veneziani, Gianluigi Zanetti, and Antonio Zorcolo.
Andrea O. Leone, Paola Marzano, Enrico Gobbetti, Riccardo Scateni, and Sergio Pedinotti.
Paola Criscione, Claudio Montani, Riccardo Scateni, and Roberto Scopigno.
www.crs4.it /vic/publications   (3052 words)

  
 CATHOLIC ENCYCLOPEDIA: Rome
Then followed the schism of Pier Leone (Anacletus II), which had hardly been ended, in 1143, when Girolamo di Pierleone, counselled by Arnold of Brescia, made Rome into a republic, modelled after the Lombard communes, under the rule of fifty-six senators.
The Gesù, connected with the professed house and general's residence of the Jesuits, is the work of Vignola (1568-73), completed by Giacomo della Porta, through the munificence of Cardinal Alessandro Farnese.
The principal fountains are: the Acqua Paola, on the Janiculum (Paul V); the Piazza S. Pietro fountain, the Tartarughe (Raphael), the Fontana del Tritone (Bernini), and, most magnificent of all, the Trevi (Clement XII, Nicolà Salvi).
www.newadvent.org /cathen/13164a.htm   (14301 words)

  
 FORWARD : Genetics   (Site not responding. Last check: 2007-10-17)
In 2001 Sontag became the third Canavan child to undergo the latest gene therapy, which consists of drilling tiny holes into different points in the brain and pumping in healthy copies of the gene into six sections of the brain.
"At that age there shouldn't be any progression — he should be in a vegetative state." Leone said she believes that a complete cure might be very close, if the money and support were there.
Watching Holovach in "As Daylight Breaks" one could definitely believe that she is the woman to rally support.
www.forward.com /issues/2003/03.08.15/genetics4.html   (784 words)

  
 CRS4 Visual Computing (ViC) / publications / 'Leone:1998:DFEc'   (Site not responding. Last check: 2007-10-17)
The aim of this research line is the study and implementation of appropriate visualization techniques for finite element data in two and three dimensions.
Such data is produced for example by chemical simulations, by fluid dynamics simulations, or, in general, anywhere high accuracy on boundary domain description is required.
Andrea O. Leone, Paola Marzano, and Enrico Gobbetti.
www.crs4.it /vic/cgi-bin/bib-page.cgi?id='Leone:1998:DFEc'   (364 words)

  
 Film shows need for NIH funding=The Hill.com=   (Site not responding. Last check: 2007-10-17)
Dr. Paola Leone, Jacob, and his parents are waiting for a five-year grant from the National Institute of Child Health and Human Development to continue Canavan research.
Jacob was one of Leone’s three patients who underwent the world’s first clinical gene therapy trial in 2001.
Experiments on Parkinson’s disease were modeled after Leone’s work.
www.hillnews.com /news/063004/nih.aspx   (396 words)

  
 With government nod, gene therapy pioneered at UNC is given to children with rare disorder
CHAPEL HILL -- A form of gene therapy created and produced at the University of North Carolina at Chapel Hill and approved by the U.S. Food and Drug Administration has been given to children with a rare, inherited neurological disorder.
This work involved collaboration with investigators at the Robert Wood Johnson Medical School, of the University of Medicine and Dentistry of New Jersey, and was led by Dr. Paola Leone and a group of 16 investigators and surgeons.
The children have Canavan disease, characterized by spongy degeneration of the brain's white matter.
www.eurekalert.org /pub_releases/2003-05/uonc-wgn053003.php   (901 words)

  
 italian genealogy,italian personal ads,italian pen pals by Tricolore.Net   (Site not responding. Last check: 2007-10-17)
However they reacted and, fortunately, after some days of searching, they found on the Internet the website of the CANAVAN FOUNDATION RESEARCH, devoted to genetic therapy for Canavan-affected children.
They found that one of the researchers in the project is an Italian PhD, Paola Leone.
They wrote her and received an immediate reply.
www.tricolore.net /cgi-bin/forum/forum.pl?reply=308   (379 words)

  
 CRS4 Visual Computing (ViC) / publications / 'Leone:1998:DFEa'   (Site not responding. Last check: 2007-10-17)
The aim of this work is the study and the implementation of appropriate visualization techniques for high-order discontinuous finite element data in two and three-dimensions.
In Proceedings 8th International Symposium on Flow Visualization, September 1998.
author = {Andrea O. Leone and Paola Marzano and Enrico Gobbetti and Riccardo Scateni and Sergio Pedinotti},
www.crs4.it /vic/cgi-bin/bib-page.cgi?id='Leone:1998:DFEa'   (362 words)

  
 Francesco Rosi
One might expect the author of Mani sulla città to have sought to confront more directly the momentous social upheaval resulting from the student protests of 1968, but with Uomini contro (1970), an adaptation of a passionate anti-war novel by Emilio Lussu, Rosi chose to go back to the time of the First World War.
With the men in the trenches demoralised and ready to give up, a struggle ensues among the officers, between the old hardliners, led by General Leone (Alain Cuny), who would continue to fight to the last man, and others led by two young lieutenants, who find themselves the "uomini contro" (”men against”) of the title.
As the logic of power plays itself out, the two younger officers are both eliminated, one dying in a final futile military assault and the other by firing squad.
www.sensesofcinema.com /contents/directors/03/rosi.html   (3770 words)

  
 Hunter's Hope: Grants Awarded
Joanne Kurtzberg, M.D. - Duke University Medical Center - Durham, NC "Selection and Screening of Umbilical Cord Blood (UCB) for Stem Cell Transplantation in Krabbe Disease"
Paola Leone, Ph.D. - University of Medicine & Dent of NJ/Robert Wood Johnson Medical School- Camden, NJ "Gene Therapy of a Rat Model of Leukodystrophy"
Paola Luzi, Ph.D. - Thomas Jefferson University - Philadelphia, PA "SV40 Containing Galactocerebrosidase (GALC) Transgene: A Novel Gene Therapy Approach for the Treatment of Krabbe Disease"
www.huntershope.org /research/grants_awarded.asp   (631 words)

  
 JeffNEWS Online... News Briefs   (Site not responding. Last check: 2007-10-17)
Just type in the appropriate season and you will learn more!
Jacob's Ladder, which is also known as The Canadian Foundation for Control of Neurodegenerative Disease, together with the generosity of its supporters, has provided a total of $80,692 to support the research of Paola Leone, PhD, Assistant Professor of Neurosurgery and Associate Director of the CNS Gene Therapy Center at Jefferson.
Leone, along with colleagues Matthew During, MD, Professor of Neurosurgery and Director of the CNS Gene Therapy Center, and Andrew Freese, MD, PhD, Associate Professor of Neurosurgery and Director of Neurosurgery Research, are progressing in their quest to develop a gene therapy treatment for Canavan disease.
www.tju.edu /jeffnews/past/00/july/briefs.html   (557 words)

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