基本信息
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职业迁徙
个人简介
Description of Research Expertise
Keywords:
Neurodegenerative Disease
RNA biology
Gene therapy
Animal models
Human treatment
Research in the Davidson Laboratory is focused on inherited genetic diseases that cause central nervous system dysfunction, with a focus on:
i) recessive, childhood onset neurodegenerative disease, such as the lysosomal storage diseases mucopolysaccharidoses and Battens disease;
ii) dominant genetic diseases, specifically the CAG repeat disorders, Huntington’s disease and spinal cerebellar ataxia;
iii) understanding how changes in the transcriptome impact neural development and neurodegenerative disease processes.
Our research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop small molecule or gene therapy based strategies for therapy. In recent work, we demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span. We continue to develop novel vector systems to improve therapeutic outcomes.
Therapies for dominant disorders are an exciting challenge and require that the dominant disease allele be silenced. To approach this, we developed reagents for expressing inhibitory RNAs or editing machinery (e.g., CrispR/Cas9 approaches) in vivo to improve disease phenotypes in relevant animal models.
Finally, we investigate how the transcriptome is altered in neurological diseases. Evaluation of splicing changes has led us to discover novel players in disease pathogenesis that include noncoding RNAs and RNA binding proteins. This work is revealing new pathways of pathogenesis and novel targets for therapy.
Keywords:
Neurodegenerative Disease
RNA biology
Gene therapy
Animal models
Human treatment
Research in the Davidson Laboratory is focused on inherited genetic diseases that cause central nervous system dysfunction, with a focus on:
i) recessive, childhood onset neurodegenerative disease, such as the lysosomal storage diseases mucopolysaccharidoses and Battens disease;
ii) dominant genetic diseases, specifically the CAG repeat disorders, Huntington’s disease and spinal cerebellar ataxia;
iii) understanding how changes in the transcriptome impact neural development and neurodegenerative disease processes.
Our research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop small molecule or gene therapy based strategies for therapy. In recent work, we demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span. We continue to develop novel vector systems to improve therapeutic outcomes.
Therapies for dominant disorders are an exciting challenge and require that the dominant disease allele be silenced. To approach this, we developed reagents for expressing inhibitory RNAs or editing machinery (e.g., CrispR/Cas9 approaches) in vivo to improve disease phenotypes in relevant animal models.
Finally, we investigate how the transcriptome is altered in neurological diseases. Evaluation of splicing changes has led us to discover novel players in disease pathogenesis that include noncoding RNAs and RNA binding proteins. This work is revealing new pathways of pathogenesis and novel targets for therapy.
研究兴趣
论文共 162 篇作者统计合作学者相似作者
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MOLECULAR THERAPYno. 4 (2023): 22-22
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Terence Gall-Duncan,Jennifer Luo,Carla-Marie Jurkovic,Laura A. Fischer,Kyota Fujita,Amit L. Deshmukh,Rachel J. Harding,Stephanie Tran,Mustafa Mehkary,Vanessa Li,David E. Leib,Ran Chen,Hikari Tanaka,Amanda G. Mason,Dominique Levesque,Mahreen Khan,Mortezaali Razzaghi,Tanya Prasolava,Stella Lanni,Nozomu Sato,Marie-Christine Caron,Gagan B. Panigrahi,Peixiang Wang,Rachel Lau,Arturo Lopez Castel,Jean-Yves Masson,Lynette Tippett,Clinton Turner,Maria Spies,Albert R. La Spad,Eric I. Campos,Maurice A. Curtis,Francois-Michel Boisvert,Richard L. M. Faull,Beverly L. Davidson,Masayuki Nakamori,Hitoshi Okazawa,Marc S. Wold,Christopher E. Pearson
Cellno. 22 (2023): 4898-4919.e25
Defne A. Amado,Alejandro Mas Monteys,Alicia R. Smith, Katherine Whiteman,Guillem Chillon Bosch,Ashley Robbins,Aleksandar Izda,Shareen Nelson, Abigail I. Dichter,Beverly L. Davidson
ANNALS OF NEUROLOGY (2023): S221-S221
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ANNALS OF NEUROLOGY (2023): S217-S218
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MOLECULAR THERAPYno. 4 (2023): 467-467
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MOLECULAR THERAPYno. 4 (2023): 666-666
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MOLECULAR THERAPYno. 4 (2023): 2-2
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ANNALS OF NEUROLOGY (2023): S272-S272
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作者统计
#Papers: 162
#Citation: 7789
H-Index: 47
G-Index: 88
Sociability: 6
Diversity: 0
Activity: 1
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