Genetic therapy for congenital myopathies

Current Opinion in Neurology ◽

10.1097/wco.0000000000000978 ◽

2021 ◽

Vol Publish Ahead of Print ◽

Author(s):

Nika Maani ◽

Sophie Karolczak ◽

James J. Dowling

Keyword(s):

Genetic Therapy ◽

Congenital Myopathies

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Stem Cells and Genetic Therapy for Hair Cell–Related Hearing Loss

10.1055/s-2009-1224357 ◽

2009 ◽

Vol 19 (S 02) ◽

Author(s):

J. Ito

Keyword(s):

Hearing Loss ◽

Hair Cell ◽

Genetic Therapy

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Faculty Opinions recommendation of RYR1 mutations are a common cause of congenital myopathies with central nuclei.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.5830956.5814054 ◽

2010 ◽

Author(s):

Nigel Laing ◽

Gianina Ravenscroft

Keyword(s):

Congenital Myopathies ◽

Common Cause ◽

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Faculty Opinions recommendation of Pharmacological treatment and BBB-targeted genetic therapy for MCT8-dependent hypomyelination in zebrafish.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.726771442.793540178 ◽

2017 ◽

Author(s):

Luca Persani

Keyword(s):

Pharmacological Treatment ◽

Genetic Therapy

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The future of ALS might move towards Genetic Therapy

Revue Neurologique ◽

10.1016/j.neurol.2021.01.007 ◽

2021 ◽

Author(s):

P. Corcia ◽

P. Couratier ◽

P. Vourc’h

Keyword(s):

Genetic Therapy ◽

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Epidermal Growth Factor Receptor as a Genetic Therapy Target for Carcinoma Cell Radiosensitization

JNCI Journal of the National Cancer Institute ◽

10.1093/jnci/93.12.921 ◽

2001 ◽

Vol 93 (12) ◽

pp. 921-929 ◽

Author(s):

G. Lammering ◽

T. H. Hewit ◽

W. T. Hawkins ◽

J. N. Contessa ◽

D. B. Reardon ◽

...

Keyword(s):

Epidermal Growth Factor Receptor ◽

Growth Factor ◽

Epidermal Growth Factor ◽

Carcinoma Cell ◽

Growth Factor Receptor ◽

Genetic Therapy ◽

Epidermal Growth ◽

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What's new in neuromuscular disorders? The congenital myopathies

European Journal of Paediatric Neurology ◽

10.1016/s1090-3798(02)00136-8 ◽

2003 ◽

Vol 7 (1) ◽

pp. 23-30 ◽

Author(s):

Heinz Jungbluth ◽

Caroline A Sewry ◽

Francesco Muntoni

Keyword(s):

Neuromuscular Disorders ◽

Congenital Myopathies

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CONGENITAL MYOPATHIES

Neuromuscular Disorders ◽

10.1016/j.nmd.2021.07.078 ◽

2021 ◽

Vol 31 ◽

pp. S65

Author(s):

L. van den Bersselaar ◽

N. Kruijt ◽

S. Buckens ◽

L. Joosten ◽

G. Scheffer ◽

...

Keyword(s):

Congenital Myopathies

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CONGENITAL MYOPATHIES: GENERAL AND RYR1

Neuromuscular Disorders ◽

10.1016/j.nmd.2018.06.077 ◽

2018 ◽

Vol 28 ◽

pp. S45

Author(s):

M. Garibaldi ◽

J. Rendu ◽

J. Brocard ◽

E. lacene ◽

M. Beuvin ◽

...

Keyword(s):

Congenital Myopathies

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CONGENITAL MYOPATHIES: GENERAL AND RYR1

Neuromuscular Disorders ◽

10.1016/j.nmd.2018.06.067 ◽

2018 ◽

Vol 28 ◽

pp. S42-S43

Author(s):

M. Schülke ◽

W. Stenzel ◽

M. Schwarz ◽

H. Goebel

Keyword(s):

Congenital Myopathies

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Novel SPEG mutations in congenital myopathies: Genotype–phenotype correlations

Muscle & Nerve ◽

10.1002/mus.26378 ◽

2018 ◽

Vol 59 (3) ◽

pp. 357-362 ◽

Author(s):

Anita E. Qualls ◽

Sandra Donkervoort ◽

Johanna C. Herkert ◽

Alissa M. D'gama ◽

Diana Bharucha‐Goebel ◽

...

Keyword(s):

Congenital Myopathies

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