Massaro G - Search Results

1

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.

Massaro G, Geard AF, Liu W, Coombe-Tennant O, Waddington SN, Baruteau J, Gissen P, Rahim AA. Massaro G, et al. Biomolecules. 2021 Apr 20;11(4):611. doi: 10.3390/biom11040611. Biomolecules. 2021. PMID: 33924076 Free PMC article. Review.

2

Perinatal systemic gene delivery using adeno-associated viral vectors.

Karda R, Buckley SM, Mattar CN, Ng J, Massaro G, Hughes MP, Kurian MA, Baruteau J, Gissen P, Chan JK, Bacchelli C, Waddington SN, Rahim AA. Karda R, et al. Among authors: massaro g. Front Mol Neurosci. 2014 Nov 14;7:89. doi: 10.3389/fnmol.2014.00089. eCollection 2014. Front Mol Neurosci. 2014. PMID: 25452713 Free PMC article. Review.

3

Delineating pathological pathways in a chemically induced mouse model of Gaucher disease.

Vardi A, Zigdon H, Meshcheriakova A, Klein AD, Yaacobi C, Eilam R, Kenwood BM, Rahim AA, Massaro G, Merrill AH Jr, Vitner EB, Futerman AH. Vardi A, et al. Among authors: massaro g. J Pathol. 2016 Aug;239(4):496-509. doi: 10.1002/path.4751. Epub 2016 Jul 5. J Pathol. 2016. PMID: 27234572

4

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease.

Hughes MP, Smith DA, Morris L, Fletcher C, Colaco A, Huebecker M, Tordo J, Palomar N, Massaro G, Henckaerts E, Waddington SN, Platt FM, Rahim AA. Hughes MP, et al. Among authors: massaro g. Hum Mol Genet. 2018 Sep 1;27(17):3079-3098. doi: 10.1093/hmg/ddy212. Hum Mol Genet. 2018. PMID: 29878115 Free PMC article.

5

Fetal gene therapy for neurodegenerative disease of infants.

Massaro G, Mattar CNZ, Wong AMS, Sirka E, Buckley SMK, Herbert BR, Karlsson S, Perocheau DP, Burke D, Heales S, Richard-Londt A, Brandner S, Huebecker M, Priestman DA, Platt FM, Mills K, Biswas A, Cooper JD, Chan JKY, Cheng SH, Waddington SN, Rahim AA. Massaro G, et al. Nat Med. 2018 Sep;24(9):1317-1323. doi: 10.1038/s41591-018-0106-7. Epub 2018 Jul 16. Nat Med. 2018. PMID: 30013199 Free PMC article.

6

Epilepsy Gene Therapy Using an Engineered Potassium Channel.

Snowball A, Chabrol E, Wykes RC, Shekh-Ahmad T, Cornford JH, Lieb A, Hughes MP, Massaro G, Rahim AA, Hashemi KS, Kullmann DM, Walker MC, Schorge S. Snowball A, et al. Among authors: massaro g. J Neurosci. 2019 Apr 17;39(16):3159-3169. doi: 10.1523/JNEUROSCI.1143-18.2019. Epub 2019 Feb 12. J Neurosci. 2019. PMID: 30755487 Free PMC article.

7

Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.

Plotegher N, Perocheau D, Ferrazza R, Massaro G, Bhosale G, Zambon F, Rahim AA, Guella G, Waddington SN, Szabadkai G, Duchen MR. Plotegher N, et al. Among authors: massaro g. Cell Death Differ. 2020 May;27(5):1588-1603. doi: 10.1038/s41418-019-0442-2. Epub 2019 Nov 4. Cell Death Differ. 2020. PMID: 31685979 Free PMC article.

8

Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease.

Kleine Holthaus SM, Herranz-Martin S, Massaro G, Aristorena M, Hoke J, Hughes MP, Maswood R, Semenyuk O, Basche M, Shah AZ, Klaska IP, Smith AJ, Mole SE, Rahim AA, Ali RR. Kleine Holthaus SM, et al. Among authors: massaro g. Hum Mol Genet. 2019 Dec 1;28(23):3867-3879. doi: 10.1093/hmg/ddz210. Hum Mol Genet. 2019. PMID: 31807779

9

Systemic AAV9 gene therapy using the synapsin I promoter rescues a mouse model of neuronopathic Gaucher disease but with limited cross-correction potential to astrocytes.

Massaro G, Hughes MP, Whaler SM, Wallom KL, Priestman DA, Platt FM, Waddington SN, Rahim AA. Massaro G, et al. Hum Mol Genet. 2020 Jul 29;29(12):1933-1949. doi: 10.1093/hmg/ddz317. Hum Mol Genet. 2020. PMID: 31919491 Free PMC article.

10

Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.

Plotegher N, Perocheau D, Ferrazza R, Massaro G, Bhosale G, Zambon F, Rahim AA, Guella G, Waddington SN, Szabadkai G, Duchen MR. Plotegher N, et al. Among authors: massaro g. Cell Death Differ. 2020 Aug;27(8):2534. doi: 10.1038/s41418-020-0525-0. Cell Death Differ. 2020. PMID: 32152554 Free PMC article.

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