Intravenous administration of CLN3 gene therapy for juvenile neuronal ceroid lipofuscinosis

An improved, novel, systemically administered AAV gene therapy for treatment of CLN3 juvenile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2018.12.202 ◽

2019 ◽

Vol 126 (2) ◽

pp. S82-S83 ◽

Cited By ~ 1

Author(s):

Scott Kerns ◽

M. Arnold ◽

A. Aldrich ◽

R. Fallet ◽

Tammy Kielian ◽

...

Keyword(s):

Gene Therapy ◽

Adeno-associated virus 9 gene therapy for juvenile neuronal ceroid lipofuscinosis

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2015.12.319 ◽

2016 ◽

Vol 117 (2) ◽

pp. S67

Author(s):

Tammy Kielian ◽

Megan E. Bosch ◽

Amy Aldrich ◽

Rachel Fallet ◽

Kevin D. Foust

Keyword(s):

Gene Therapy ◽

Adeno Associated Virus ◽

218. Gene Therapy and Enzyme Replacement in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

Molecular Therapy ◽

10.1016/j.ymthe.2006.08.243 ◽

2006 ◽

Vol 13 ◽

pp. S84

Author(s):

Michael Chang ◽

David Sleat ◽

Seng Cheng ◽

Marco Passini ◽

Peter Lobel ◽

...

Keyword(s):

Gene Therapy ◽

Mouse Model ◽

Infantile Neuronal Ceroid Lipofuscinosis ◽

Enzyme Replacement ◽

Loss of pigment-laden stellate cells: A severe alteration of the isocortex in juvenile neuronal ceroid-lipofuscinosis

Acta Neuropathologica ◽

10.1007/bf01273267 ◽

1978 ◽

Vol 42 (1) ◽

pp. 53-57 ◽

Cited By ~ 47

Author(s):

H. Braak ◽

H. H. Goebel

Keyword(s):

Stellate Cells ◽

Photoreceptor phagosome processing defects and disturbed autophagy in retinal pigment epithelium ofCln3Δex1-6mice modelling juvenile neuronal ceroid lipofuscinosis (Batten disease)

Human Molecular Genetics ◽

10.1093/hmg/ddv406 ◽

2015 ◽

pp. ddv406 ◽

Cited By ~ 7

Author(s):

Silène T. Wavre-Shapton ◽

Alessandra A. Calvi ◽

Mark Turmaine ◽

Miguel C. Seabra ◽

Daniel F. Cutler ◽

...

Keyword(s):

Retinal Pigment Epithelium ◽

Pigment Epithelium ◽

Retinal Pigment ◽

Batten Disease ◽

Ceroid Lipofuscinosis ◽

Processing Defects

Altered Flurothyl Seizure Induction Latency, Phenotype, and Subsequent Mortality in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis/Batten Disease

Epilepsia ◽

10.1046/j.1528-1157.2002.16002.x ◽

2002 ◽

Vol 43 (10) ◽

pp. 1137-1140 ◽

Cited By ~ 11

Author(s):

Elizabeth Kriscenski-Perry ◽

Craig D. Applegate ◽

Andrew Serour ◽

Timothy R. Mhyre ◽

Christopher C. Leonardo ◽

...

Keyword(s):

Mouse Model ◽

Batten Disease ◽

Diagnostic Value of Electron Microscopy in a Case of Juvenile Neuronal Ceroid Lipofuscinosis

Ultrastructural Pathology ◽

10.1080/019131201753136296 ◽

2001 ◽

Vol 25 (4) ◽

pp. 285-288 ◽

Cited By ~ 5

Author(s):

Birgitta Carlén, Elisabet Englund

Keyword(s):

Electron Microscopy ◽

Diagnostic Value ◽

Immunomodulation with minocycline rescues retinal degeneration in juvenile neuronal ceroid lipofuscinosis mice highly susceptible to light damage

Disease Models & Mechanisms ◽

10.1242/dmm.033597 ◽

2018 ◽

Vol 11 (9) ◽

pp. dmm033597 ◽

Cited By ~ 9

Author(s):

Katharina Dannhausen ◽

Christoph Möhle ◽

Thomas Langmann

Keyword(s):

Retinal Degeneration ◽

Light Damage ◽

Psychiatric symptoms of children and adolescents with juvenile neuronal ceroid lipofuscinosis

Journal of Intellectual Disability Research ◽

10.1111/j.1365-2788.2005.00659.x ◽

2005 ◽

Vol 49 (1) ◽

pp. 25-32 ◽

Cited By ~ 24

Author(s):

M. L. Backman ◽

P. R. Santavuori ◽

L. E. Aberg ◽

E. T. Aronen

Keyword(s):

Children And Adolescents ◽

Psychiatric Symptoms ◽

Comparison of the Clinical Courses in Patients with Juvenile Neuronal Ceroid Lipofuscinosis Receiving Antioxidant Treatment and Those without Antioxidant Treatment

Lipofuscin and Ceroid Pigments - Advances in Experimental Medicine and Biology ◽

10.1007/978-1-4899-5339-1_19 ◽

1990 ◽

pp. 273-282 ◽

Cited By ~ 3

Author(s):

P. Santavuori ◽

H. Heiskala ◽

T. Autti ◽

E. Johansson ◽

T. Westermarck

Keyword(s):

Antioxidant Treatment ◽