DOI: 10.1038/sj.ejhg.5201102. Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy

Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy

10.1038/sj.ejhg.5201102

Crossref journal-article

Springer Science and Business Media LLC

European Journal of Human Genetics (297)

Bibliography

Andreassi, C., Angelozzi, C., Tiziano, F. D., Vitali, T., De Vincenzi, E., Boninsegna, A., Villanova, M., Bertini, E., Pini, A., Neri, G., & Brahe, C. (2003). Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy. European Journal of Human Genetics, 12(1), 59â65.

Authors 11

Catia Andreassi (first)
Carla Angelozzi (additional)
Francesco D Tiziano (additional)
Tiziana Vitali (additional)
Eleonora De Vincenzi (additional)
Alma Boninsegna (additional)
Marcello Villanova (additional)
Enrico Bertini (additional)
Antonella Pini (additional)
Giovanni Neri (additional)
Christina Brahe (additional)

References 29 Referenced 205

Lefebvre S, Bürglen L, Reboullet S et al: Identification and characterization of a spinal muscular atrophy-determining gene. Cell 1995; 80: 155–165. (10.1016/0092-8674(95)90460-3) / Cell by S Lefebvre (1995)
Lorson CL, Hahnen E, Androphy EJ, Wirth B : A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc Natl Acad Sci USA 1999; 96: 6307–6311. (10.1073/pnas.96.11.6307) / Proc Natl Acad Sci USA by CL Lorson (1999)
Monani UR, Lorson CL, Parsons DW et al: A single nucleotide difference that alters splicing patterns distinguishes the SMA gene SMN1 from the copy gene SMN. Hum Mol Genet 1999; 8: 1177–1183. (10.1093/hmg/8.7.1177) / Hum Mol Genet by UR Monani (1999)
McAndrew PE, Parsons DW, Simard LR et al: Identification of proximal spinal muscular atrophy carriers and patients by analysis of SMNt and SMNc gene copy number. Am J Hum Genet 1997; 69: 1411–1422. (10.1086/515465) / Am J Hum Genet by PE McAndrew (1997)
Vitali T, Sossi V, Tiziano F et al: Detection of the survival motor neuron (SMN) genes by FISH: further evidence for a role of SMN2 in the modulation of disease severity in SMA patients. Hum Mol Genet 1999; 8: 2525–2532. (10.1093/hmg/8.13.2525) / Hum Mol Genet by T Vitali (1999)
Brahe C : Copies of the survival motor neuron gene in spinal muscular atrophy: the more, the better. Neuromusc Disord 2000; 10: 274–275. (10.1016/S0960-8966(99)00137-6) / Neuromusc Disord by C Brahe (2000)
Feldkötter M, Schwarzer V, Wirth R, Wienker TI, Wirth B : Quantitative analysis of SMN1 and SMN2 based on real-time LightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy. Am J Hum Genet 2002; 70: 358–368. (10.1086/338627) / Am J Hum Genet by M Feldkötter (2002)
Liu Q, Dreyfuss G : A novel nuclear structure containing the survival of motor neurons protein. EMBO J 1996; 15: 3555–3565. (10.1002/j.1460-2075.1996.tb00725.x) / EMBO J by Q Liu (1996)
Lefebvre S, Burlet P, Liu Q et al: Correlation between severity and SMN protein level in spinal muscular atrophy. Nat Genet 1997; 16: 265–269. (10.1038/ng0797-265) / Nat Genet by S Lefebvre (1997)
Coovert DD, Le TT, McAndrew PE et al: The survival motor neuron protein in spinal muscular atrophy. Hum Mol Genet 1997; 6: 1205–1214. (10.1093/hmg/6.8.1205) / Hum Mol Genet by DD Coovert (1997)
Patrizi AL, Tiziano F, Zappata S, Donati A, Neri G, Brahe C : SMN protein analysis in fibroblast, amniocyte, and CVS cultures from spinal muscular atrophy patients and its relevance for diagnosis. Eur J Hum Genet 1999; 7: 301–309. (10.1038/sj.ejhg.5200286) / Eur J Hum Genet by AL Patrizi (1999)
Miller RG, Moore DH, Dronsky V et al: A placebo-controlled trial of gabapentin in spinal muscular atrophy. J Neurol Sci 2001; 191: 127–131. (10.1016/S0022-510X(01)00632-3) / J Neurol Sci by RG Miller (2001)
Tzeng AC, Cheng J, Fryczynski H et al: A study of thyrotropin-releasing hormone for the treatment of spinal muscular atrophy: a preliminary report. Am J Phys Med Rehabil 2000; 79: 435–440. (10.1097/00002060-200009000-00005) / Am J Phys Med Rehabil by AC Tzeng (2000)
Kinali M, Mercuri E, Main M et al: Pilot trial of albuterol in spinal muscular atrophy. Neurology 2002; 59: 609–610. (10.1212/WNL.59.4.609) / Neurology by M Kinali (2002)
Baron-Delage S, Abadie A, Echaniz-Laguna A, Melki J, Beretta L : Interferons and IRF-1 induce expression of the survival motor neuron (SMN) genes. Mol Med 2000; 6: 957–968. (10.1007/BF03401830) / Mol Med by S Baron-Delage (2000)
Andreassi C, Jarecki J, Zhou J et al: Aclarubicin treatment restores SMN levels to cells derived from type I spinal muscular atrophy patients. Hum Mol Genet 2001; 10: 2841–2849. (10.1093/hmg/10.24.2841) / Hum Mol Genet by C Andreassi (2001)
Chang J-G, Hsieh-Li H-M, Jong Y-J, Wang NM, Tsai C-H, Li H : Treatment of spinal muscular atrophy by sodium butyrate. Proc Natl Acad Sci USA 2001; 98: 9808–9813. (10.1073/pnas.171105098) / Proc Natl Acad Sci USA by J-G Chang (2001)
Vitali T, Tiziano F, Neri G, Brahe C : SMN2 gene activation by sodium butyrate and acetyl-L-carnitine in cell cultures from SMA patients; Abstract, Fifth Annual North American/International Spinal Muscular Atrophy Research and Clinical Meeting; June 22–24, 2001; Chicago.
Tiziano FD, Andreassi C, Angelozzi C et al: Upregulation of SMN2 gene expression by 4-phenylbutyrate: relevance for a therapeutic approach to spinal muscular atrophy. Am J Hum Genet 2002; 71 (Suppl): A2445. / Am J Hum Genet by FD Tiziano (2002)
Maestri NE, Brusilow SW, Clissold DB, Bassett S : Long term treatment of girls with ornithine transcarbamylase deficiency. N Engl J Med 1996; 335: 855–859. (10.1056/NEJM199609193351204) / N Engl J Med by NE Maestri (1996)
Gilbert J, Baker SD, Bowling MK et al: A phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies. Clin Cancer Res 2001; 7: 2292–2300. / Clin Cancer Res by J Gilbert (2001)
Davis T, Kennedy C, Chiew Y-E, Clarke CL, deFazio A : Histone deacetylase inhibitors decrease proliferation and modulate cell cycle gene expression in normal mammary epithelial cells. Clin Cancer Res 2000; 6: 4334–4342. / Clin Cancer Res by T Davis (2000)
McGrath-Morrow SA, Stahl JL : G1 phase growth arrest and induction of p21waf1/Cip/Sdi1 in IB3-1 cells treated with 4-sodium phenylbutyrate. J Pharmacol Exp Therap 2000; 294: 941–947. / J Pharmacol Exp Therap by SA McGrath-Morrow (2000)
Dover GJ, Brusilow S, Charache S : Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium pehylbutyrate. Blood 1994; 84: 339–343. (10.1182/blood.V84.1.339.339) / Blood by GJ Dover (1994)
Gore SD, Samid D, Weng L-J : Impact of the putative differentiating agents sodium phenylbutyrate and sodium phenylacetate on proliferation, differentiation, and apoptosis of primary neoplastic myeloid cells. Clin Cancer Res 1997; 3: 1755–1762. / Clin Cancer Res by SD Gore (1997)
Carducci MA, Nelson JB, Chantack KM et al: Phenylbutyrate induces apoptosis in human prostate cancer and is more potent than phenylacetate. Clin Cancer Res 1996; 2: 379–387. / Clin Cancer Res by MA Carducci (1996)
Brusilow SW, Maestri NE : Urea cycle disorders: diagnosis, pathophysiology and therapy. Adv Pediat 1996; 23: 127–170. (10.1016/S0065-3101(24)00072-0) / Adv Pediat by SW Brusilow (1996)
Kemp S, Wei H-M, Lu J-F et al: Gene redundancy of pharmacological gene therapy: implications for X-linked adrenoleukodystrophy. Nat Med 1998; 4: 1261–1268. (10.1038/3242) / Nat Med by S Kemp (1998)
Berg S, Serabe B, Aleksic A et al: Pharmacokinetics and cerebrospinal fluid penetration of phenylacetate and phenylbutyrate in the nonhuman primate. Cancer Chemother Pharmacol 2001; 47: 385–390. (10.1007/s002800000256) / Cancer Chemother Pharmacol by S Berg (2001)

Dates

Type	When
Created	21 years, 10 months ago (Oct. 15, 2003, 5:06 a.m.)
Deposited	1 year, 7 months ago (Jan. 10, 2024, 7:39 p.m.)
Indexed	4 weeks, 1 day ago (Aug. 7, 2025, 4:33 p.m.)
Issued	21 years, 10 months ago (Oct. 22, 2003)
Published	21 years, 10 months ago (Oct. 22, 2003)
Published Online	21 years, 10 months ago (Oct. 22, 2003)
Published Print	21 years, 8 months ago (Jan. 1, 2004)

Funders 0

None

BibTeX

@article{Andreassi_2003, title={Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy}, volume={12}, ISSN={1476-5438}, url={http://dx.doi.org/10.1038/sj.ejhg.5201102}, DOI={10.1038/sj.ejhg.5201102}, number={1}, journal={European Journal of Human Genetics}, publisher={Springer Science and Business Media LLC}, author={Andreassi, Catia and Angelozzi, Carla and Tiziano, Francesco D and Vitali, Tiziana and De Vincenzi, Eleonora and Boninsegna, Alma and Villanova, Marcello and Bertini, Enrico and Pini, Antonella and Neri, Giovanni and Brahe, Christina}, year={2003}, month=oct, pages={59–65} }

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