Kaspar B - Search Results

1

Adeno Associated Virus 9-Based Gene Therapy Delivers a Functional Monocarboxylate Transporter 8, Improving Thyroid Hormone Availability to the Brain of Mct8-Deficient Mice.

Iwayama H, Liao XH, Braun L, Bárez-López S, Kaspar B, Weiss RE, Dumitrescu AM, Guadaño-Ferraz A, Refetoff S. Iwayama H, et al. Among authors: kaspar b. Thyroid. 2016 Sep;26(9):1311-9. doi: 10.1089/thy.2016.0060. Epub 2016 Aug 23. Thyroid. 2016. PMID: 27432638 Free PMC article.

2

AAV9-MCT8 Delivery at Juvenile Stage Ameliorates Neurological and Behavioral Deficits in a Mouse Model of MCT8-Deficiency.

Liao XH, Avalos P, Shelest O, Ofan R, Shilo M, Bresee C, Likhite S, Vit JP, Heuer H, Kaspar B, Meyer K, Dumitrescu AM, Refetoff S, Svendsen CN, Vatine GD. Liao XH, et al. Among authors: kaspar b. Thyroid. 2022 Jul;32(7):849-859. doi: 10.1089/thy.2022.0034. Epub 2022 May 20. Thyroid. 2022. PMID: 35350867 Free PMC article.

3

Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.

Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Meyer K, et al. Mol Ther. 2015 Mar;23(3):477-87. doi: 10.1038/mt.2014.210. Epub 2014 Oct 31. Mol Ther. 2015. PMID: 25358252 Free PMC article.

4

Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders.

Bevan AK, Duque S, Foust KD, Morales PR, Braun L, Schmelzer L, Chan CM, McCrate M, Chicoine LG, Coley BD, Porensky PN, Kolb SJ, Mendell JR, Burghes AH, Kaspar BK. Bevan AK, et al. Mol Ther. 2011 Nov;19(11):1971-80. doi: 10.1038/mt.2011.157. Epub 2011 Aug 2. Mol Ther. 2011. PMID: 21811247 Free PMC article.

5

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.

Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Foust KD, et al. Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28. Nat Biotechnol. 2010. Retraction in: Nat Biotechnol. 2022 Nov;40(11):1692. doi: 10.1038/s41587-022-01497-7 PMID: 20190738 Free PMC article. Retracted.

6

Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.

Foust KD, Salazar DL, Likhite S, Ferraiuolo L, Ditsworth D, Ilieva H, Meyer K, Schmelzer L, Braun L, Cleveland DW, Kaspar BK. Foust KD, et al. Mol Ther. 2013 Dec;21(12):2148-59. doi: 10.1038/mt.2013.211. Epub 2013 Sep 6. Mol Ther. 2013. PMID: 24008656 Free PMC article.

7

Additive amelioration of ALS by co-targeting independent pathogenic mechanisms.

Frakes AE, Braun L, Ferraiuolo L, Guttridge DC, Kaspar BK. Frakes AE, et al. Ann Clin Transl Neurol. 2017 Jan 11;4(2):76-86. doi: 10.1002/acn3.375. eCollection 2017 Feb. Ann Clin Transl Neurol. 2017. PMID: 28168207 Free PMC article.

8

A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, Al-Zaidy S, Govoni A, Rodino-Klapac LR, Hogan MJ, Kaspar BK. Mendell JR, et al. Mol Ther. 2015 Jan;23(1):192-201. doi: 10.1038/mt.2014.200. Epub 2014 Oct 17. Mol Ther. 2015. PMID: 25322757 Free PMC article. Clinical Trial.

9

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK. Mendell JR, et al. N Engl J Med. 2017 Nov 2;377(18):1713-1722. doi: 10.1056/NEJMoa1706198. N Engl J Med. 2017. PMID: 29091557 Free article. Clinical Trial.

10

Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.

Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Petruska JC, Lucchesi PA, Burghes AH, Kaspar BK. Bevan AK, et al. Hum Mol Genet. 2010 Oct 15;19(20):3895-905. doi: 10.1093/hmg/ddq300. Epub 2010 Jul 16. Hum Mol Genet. 2010. PMID: 20639395 Free PMC article.

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