Christiano Alves
Dr. Christiano Alves is an Assistant Professor of Pathology at the University of Pittsburgh School of Medicine. His research combines genome engineering, neuroscience, and translational medicine to develop next-generation CRISPR technologies for the treatment of severe neurological and metabolic diseases. Through innovative research, interdisciplinary collaboration, and a strong commitment to mentorship, his laboratory aims to accelerate the translation of scientific discoveries into life-changing therapies
- Alves, C.R.R., & Kleinstiver, B.P. (2026). Engineering of a bespoke CRISPR base editor to treat a severe vascular disease. Nat Biomed Eng, 10(5), 850-851.Springer Nature. doi: 10.1038/s41551-025-01510-9.
- Alves, C.R.R., Das, S., Krishnan, V., Ha, L.L., Fox, L.R., Stutzman, H.E., Shamber, C.E., Kalailingam, P., McCarthy, S., Lino Cardenas, C.L., Fong, C.E., Imai, T., Mitra, S., Yun, S., Wood, R.K., Benning, F.M.C., Roh, K., Lawton, J., Kim, N., Silverstein, R.A., Ferreira da Silva, J., de la Cruz, D., Richa, R., Xie, J., Gray-Edwards, H.L., Malhotra, R., Chung, D.Y., Chao, L.H., Tsai, S.Q., Maguire, C.A., Lindsay, M.E., Kleinstiver, B.P., & Musolino, P.L. (2026). Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice. Nat Biomed Eng, 10(5), 952-967.Springer Nature. doi: 10.1038/s41551-025-01499-1.
- Garner, R., Ha, L.L., Nery, F.C., Spellman, R.G., Chehade, L., Eichelberger, E.J., Da Silva Duarte Lepez, S., Johnstone, A.J., Kothary, R., Swoboda, K.J., & Alves, C.R.R. (2026). Cardiac defects in spinal muscular atrophy and the role of SMN in cardiomyocyte homeostasis. bioRxiv, 2026.03.20.713246.Cold Spring Harbor Laboratory. doi: 10.64898/2026.03.20.713246.
- Singh, R.N., & Alves, C.R.R. (2026). Harnessing CRISPR-dCas13Rx to identify novel antisense targets for therapeutic splicing modulation. Mol Ther Nucleic Acids, 37(2), 102895.Elsevier. doi: 10.1016/j.omtn.2026.102895.
- Spellman, R.G., Ha, L.L., Da Silva Duarte Lepez, S., Arruda, E.A., Rodrigues, E., Swoboda, K.J., & Alves, C.R.R. (2026). Early life safety profiling of gene therapy for spinal muscular atrophy. Gene Ther, 33(1), 89-96.Springer Nature. doi: 10.1038/s41434-025-00529-6.
- Padget, M., Sörme, P., Spezia, M., Slaugenhaupt, S.A., McKowen, J., Kirchner, E., Ameral, N., Reis, S.A., Slater, D., Haggarty, S.J., Kleinstiver, B.P., Dunn, E.C., Larocque, R., Alves, C.R.R., Stern, M., Pelli, A., & Duhaime, A.C. (2025). Environmental impact, cost, and acceptability of a laboratory sustainability certification program for biomedical research in an academic medical center. J Clim Chang Health, 23, 100401.Elsevier. doi: 10.1016/j.joclim.2024.100401.
- Pigini, P., Xu, H., Ji, Y., Lindmeier, H., Saltzman, H.R., Yun, S., Alves, C.R.R., Silva, M.C., Gao, D., & Morini, E. (2025). Poison exon splicing in the human brain: a new paradigm for understanding and targeting neurological disorders. In bioRxiv. doi: 10.1101/2025.06.26.661736.
- Alves, C.R.R., Das, S., Krishnan, V., Ha, L.L., Fox, L.R., Stutzman, H.E., Shamber, C.E., Kalailingam, P., McCarthy, S., Lino Cardenas, C.L., Fong, C.E., Imai, T., Mitra, S., Yun, S., Wood, R.K., Benning, F.M.C., Lawton, J., Kim, N., Silverstein, R.A., da Silva, J.F., de la Cruz, D., Richa, R., Malhotra, R., Chung, D.Y., Chao, L.H., Tsai, S.Q., Maguire, C.A., Lindsay, M.E., Kleinstiver, B.P., & Musolino, P.L. (2024). In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor. In bioRxiv. doi: 10.1101/2024.11.11.621817.
- Alves, C.R.R., Ha, L.L., Yaworski, R., Sutton, E.R., Lazzarotto, C.R., Christie, K.A., Reilly, A., Beauvais, A., Doll, R.M., de la Cruz, D., Maguire, C.A., Swoboda, K.J., Tsai, S.Q., Kothary, R., & Kleinstiver, B.P. (2024). Optimization of base editors for the functional correction of SMN2 as a treatment for spinal muscular atrophy. Nat Biomed Eng, 8(2), 118-131.Springer Nature. doi: 10.1038/s41551-023-01132-z.
- das Neves, W., Alves, C.R.R., Dos Santos, G., Alves, M.J.N.N., Deik, A., Pierce, K., Dennis, C., Buckley, L., Clish, C.B., Swoboda, K.J., Brum, P.C., & de Castro Junior, G. (2024). Physical performance and plasma metabolic profile as potential prognostic factors in metastatic lung cancer patients. Eur J Clin Invest, 54(11), e14288.Wiley. doi: 10.1111/eci.14288.
- Spellman, R.G., Ha, L.L., Da Silva Duarte Lepez, S., Arruda, E.A., Rodrigues, E., Swoboda, K.J., & Alves, C.R.R. (2024). Early Life Safety Profiling of Gene Therapy for Spinal Muscular Atrophy: A Case Series Analysis. In medRxiv. doi: 10.1101/2024.11.04.24316576.
- Yun, S., Chekuri, A., Art, J., Kondabolu, K., Slaugenhaupt, S.A., Zeltner, N., Kleinstiver, B.P., Morini, E., & Alves, C.R.R. (2024). Engineered CRISPR-Base Editors as a Permanent Treatment for Familial Dysautonomia. In bioRxiv. doi: 10.1101/2024.11.27.625322.
- Alves, C.R.R., Ha, L.L., Yaworski, R., Lazzarotto, C.R., Christie, K.A., Reilly, A., Beauvais, A., Doll, R.M., de la Cruz, D., Maguire, C.A., Swoboda, K.J., Tsai, S.Q., Kothary, R., & Kleinstiver, B.P. (2023). Base editing as a genetic treatment for spinal muscular atrophy. In bioRxiv. doi: 10.1101/2023.01.20.524978.
- da Silva Rodrigues, G., Noronha, N.Y., Almeida, M.L., Sobrinho, A.C.D.S., Watanabe, L.M., Pinhel, M.A.D.S., de Lima, J.G.R., Zhang, R., Nonino, C.B., Alves, C.R.R., & Bueno Júnior, C.R. (2023). Exercise training modifies the whole blood DNA methylation profile in middle-aged and older women. J Appl Physiol (1985), 134(3), 610-621.American Physiological Society. doi: 10.1152/japplphysiol.00237.2022.
- Neves, W.D., Alves, C.R.R., dos Santos, G., Alves, M.J.N., Deik, A., Pierce, K., Dennis, C., Buckley, L., Clish, C.B., Swoboda, K.J., Brum, P.C., & de Castro, G. (2023). Circulating metabolites and physical performance are predictors of overall survival in metastatic lung cancer patients. In medRxiv. doi: 10.1101/2023.08.23.23294489.
- Tobias, G.C., Gomes, J.L.P., Fernandes, L.G., Voltarelli, V.A., de Almeida, N.R., Jannig, P.R., de Souza, R.W.A., Negrão, C.E., Oliveira, E.M., Chammas, R., Alves, C.R.R., & Brum, P.C. (2023). Aerobic exercise training mitigates tumor growth and cancer-induced splenomegaly through modulation of non-platelet platelet factor 4 expression. Sci Rep, 13(1), 21970.Springer Nature. doi: 10.1038/s41598-023-47217-2.
- Alves, C., Goodyear, L., & Brum, P. (2022). Muscle metabolic stress determines cancer cachexia severity in mice. Front Physiol, 13, 1033932.Frontiers. doi: 10.3389/fphys.2022.1033932.
- Farrar, M.A., Groen, E., & Alves, C.R.R. (2022). Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy. Mol Ther Methods Clin Dev, 26, 96-97.Elsevier. doi: 10.1016/j.omtm.2022.06.005.
- Kusuyama, J., Makarewicz, N.S., Albertson, B.G., Alves-Wagner, A.B., Conlin, R.H., Prince, N.B., Alves, C.R.R., Ramachandran, K., Kozuka, C., Xiudong, Y., Xia, Y., Hirshman, M.F., Hatta, T., Nagatomi, R., Nozik, E.S., & Goodyear, L.J. (2022). Maternal Exercise-Induced SOD3 Reverses the Deleterious Effects of Maternal High-Fat Diet on Offspring Metabolism Through Stabilization of H3K4me3 and Protection Against WDR82 Carbonylation. Diabetes, 71(6), 1170-1181.American Diabetes Association. doi: 10.2337/db21-0706.
- Magen, I., Aharoni, S., Yacovzada, N.S., Tokatly Latzer, I., Alves, C.R.R., Sagi, L., Fattal-Valevski, A., Swoboda, K.J., Katz, J., Bruckheimer, E., Nevo, Y., & Hornstein, E. (2022). Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients. Eur J Neurol, 29(8), 2420-2430.Wiley. doi: 10.1111/ene.15382.
- Alves, C.R.R., Eichelberger, E.J., das Neves, W., Ribeiro, M.A.C., Bechara, L.R.G., Voltarelli, V.A., de Almeida, N.R., Hagen, L., Sharma, A., Ferreira, J.C.B., Swoboda, K.J., Slupphaug, G., & Brum, P.C. (2021). Cancer-induced muscle atrophy is determined by intrinsic muscle oxidative capacity. FASEB J, 35(7), e21714.Wiley. doi: 10.1096/fj.202100263R.
- Alves, C.R.R., Petrillo, M., Spellman, R., Garner, R., Zhang, R., Kiefer, M., Simeone, S., Sohn, J., Eichelberger, E.J., Rodrigues, E., Arruda, E.A., Townsend, E.L., Farwell, W., & Swoboda, K.J. (2021). Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series. Mol Ther Methods Clin Dev, 23, 524-538.Elsevier. doi: 10.1016/j.omtm.2021.10.011.
- das Neves, W., Alves, C.R.R., de Souza Borges, A.P., & de Castro, G. (2021). Serum Creatinine as a Potential Biomarker of Skeletal Muscle Atrophy in Non-small Cell Lung Cancer Patients. Front Physiol, 12, 625417.Frontiers. doi: 10.3389/fphys.2021.625417.
- Eichelberger, E.J., Alves, C.R.R., Zhang, R., Petrillo, M., Cullen, P., Farwell, W., Hurt, J.A., Staropoli, J.F., & Swoboda, K.J. (2021). Increased systemic HSP70B levels in spinal muscular atrophy infants. Ann Clin Transl Neurol, 8(7), 1495-1501.Wiley. doi: 10.1002/acn3.51377.
- Magen, I., Aharoni, S., Yacovzada, N.S., Latzer, I.T., Alves, C.R.R., Sagi, L., Fattal-Valevski, A., Swoboda, K.J., Katz, J., Bruckheimer, E., Nevo, Y., & Hornstein, E. (2021). Muscle microRNAs in the cerebrospinal fluid predict clinical response to nusinersen therapy in type II and type III spinal muscular atrophy patients. In medRxiv. doi: 10.1101/2021.07.29.21261322.
- Nigro, P., Middelbeek, R.J.W., Alves, C.R.R., Rovira-Llopis, S., Ramachandran, K., Rowland, L.A., Møller, A.B., Takahashi, H., Alves-Wagner, A.B., Vamvini, M., Makarewicz, N.S., Albertson, B.G., Hirshman, M.F., & Goodyear, L.J. (2021). Exercise Training Promotes Sex-Specific Adaptations in Mouse Inguinal White Adipose Tissue. Diabetes, 70(6), 1250-1264.American Diabetes Association. doi: 10.2337/db20-0790.
- Alves, C.R.R., Neves, W.D., de Almeida, N.R., Eichelberger, E.J., Jannig, P.R., Voltarelli, V.A., Tobias, G.C., Bechara, L.R.G., de Paula Faria, D., Alves, M.J.N., Hagen, L., Sharma, A., Slupphaug, G., Moreira, J.B.N., Wisloff, U., Hirshman, M.F., Negrão, C.E., de Castro, G., Chammas, R., Swoboda, K.J., Ruas, J.L., Goodyear, L.J., & Brum, P.C. (2020). Exercise training reverses cancer-induced oxidative stress and decrease in muscle COPS2/TRIP15/ALIEN. Mol Metab, 39, 101012.Elsevier. doi: 10.1016/j.molmet.2020.101012.
- Alves, C.R.R., Zhang, R., Johnstone, A.J., Garner, R., Eichelberger, E.J., Lepez, S.D.S.D., Yi, V., Stevens, V., Poxson, R., Schwartz, R., Zaworski, P., & Swoboda, K.J. (2020). Whole blood survival motor neuron protein levels correlate with severity of denervation in spinal muscular atrophy. Muscle Nerve, 62(3), 351-357.Wiley. doi: 10.1002/mus.26995.
- Alves, C.R.R., Zhang, R., Johnstone, A.J., Garner, R., Nwe, P.H., Siranosian, J.J., & Swoboda, K.J. (2020). Serum creatinine is a biomarker of progressive denervation in spinal muscular atrophy. Neurology, 94(9), e921-e931.Wolters Kluwer. doi: 10.1212/WNL.0000000000008762.
- Eichelberger, E.J., Alves, C.R.R., Zhang, R., Petrillo, M., Cullen, P., Farwell, W., Hurt, J.A., Staropoli, J.F., & Swoboda, K.J. (2020). Increased Systemic HSP70B Levels in Spinal Muscular Atrophy Infants. In medRxiv. doi: 10.1101/2020.11.20.20235325.
- Siranosian, J.J., Nery, F.C., Alves, C.R.R., Siranosian, B.A., Lyons, N.J., Eichelberger, E.J., Garner, R., Da Silva Duarte Lepez, S., Johnstone, A.J., Subramanian, A., & Swoboda, K.J. (2020). Whole-blood dysregulation of actin-cytoskeleton pathway in adult spinal muscular atrophy patients. Ann Clin Transl Neurol, 7(7), 1158-1165.Wiley. doi: 10.1002/acn3.51092.
- Alves, C.R.R. (2019). Exercise training: thinking ahead to counteract systemic manifestations of spinal muscular atrophy. J Physiol, 597(24), 5757-5758.Wiley. doi: 10.1113/JP279033.
- Alves, C.R.R., MacDonald, T.L., Nigro, P., Pathak, P., Hirshman, M.F., Goodyear, L.J., & Lessard, S.J. (2019). Reduced sucrose nonfermenting AMPK-related kinase (SNARK) activity aggravates cancer-induced skeletal muscle wasting. Biomed Pharmacother, 117, 109197.Elsevier. doi: 10.1016/j.biopha.2019.109197.
- Nery, F.C., Siranosian, J.J., Rosales, I., Deguise, M.O., Sharma, A., Muhtaseb, A.W., Nwe, P., Johnstone, A.J., Zhang, R., Fatouraei, M., Huemer, N., Alves, C.R.R., Kothary, R., & Swoboda, K.J. (2019). Impaired kidney structure and function in spinal muscular atrophy. Neurol Genet, 5(5), e353.Wolters Kluwer. doi: 10.1212/NXG.0000000000000353.
- Takahashi, H., Alves, C.R.R., Stanford, K.I., Middelbeek, R.J.W., Nigro, P., Ryan, R.E., Xue, R., Sakaguchi, M., Lynes, M.D., So, K., Mul, J.D., Lee, M.Y., Balan, E., Pan, H., Dreyfuss, J.M., Hirshman, M.F., Azhar, M., Hannukainen, J.C., Nuutila, P., Kalliokoski, K.K., Nielsen, S., Pedersen, B.K., Kahn, C.R., Tseng, Y.H., & Goodyear, L.J. (2019). TGF-β2 is an exercise-induced adipokine that regulates glucose and fatty acid metabolism. Nat Metab, 1(2), 291-303.Springer Nature. doi: 10.1038/s42255-018-0030-7.
- Alves, C.R.R., Faria, D.D.P., Carneiro, C.D.G., Garcez, A.T., Gutierrez, V.P., das Neves, W., de Almeida, N.R., Cury, Y., Chammas, R., & Brum, P.C. (2018). 18F-Fluoride PET/CT and 99mTc-MDP SPECT/CT can detect bone cancer at early stage in rodents. Life Sci, 206, 29-34.Elsevier. doi: 10.1016/j.lfs.2018.05.030.
- Souza, R.W.A., Alves, C.R.R., Medeiros, A., Rolim, N., Silva, G.J.J., Moreira, J.B.N., Alves, M.N., Wohlwend, M., Gebriel, M., Hagen, L., Sharma, A., Koch, L.G., Britton, S.L., Slupphaug, G., Wisløff, U., & Brum, P.C. (2018). Differential regulation of cysteine oxidative post-translational modifications in high and low aerobic capacity. Sci Rep, 8(1), 17772.Springer Nature. doi: 10.1038/s41598-018-35728-2.
- das Neves, W., de Oliveira, L.F., da Silva, R.P., Alves, C.R.R., & Lancha, A.H. (2017). Fasting: a major limitation for resistance exercise training effects in rodents. Braz J Med Biol Res, 51(1), e5427.FapUNIFESP (SciELO). doi: 10.1590/1414-431X20175427.
- Jannig, P.R., Alves, C.R.R., Voltarelli, V.A., Bozi, L.H.M., Vieira, J.S., Brum, P.C., & Bechara, L.R.G. (2017). Effects of N-acetylcysteine on isolated skeletal muscle contractile properties after an acute bout of aerobic exercise. Life Sci, 191, 46-51.Elsevier. doi: 10.1016/j.lfs.2017.10.012.
- das Neves, W., Alves, C.R.R., de Almeida, N.R., Guimarães, F.L.R., Ramires, P.R., Brum, P.C., & Lancha, A.H. (2016). Loss of strength capacity is associated with mortality, but resistance exercise training promotes only modest effects during cachexia progression. Life Sci, 163, 11-22.Elsevier. doi: 10.1016/j.lfs.2016.08.025.
- Alves, C.R.R., Borelli, M.T.C., Paineli, V.D.S., Azevedo, R.D.A., Borelli, C.C.G., Lancha Junior, A.H., Gualano, B., & Artioli, G.G. (2015). Development of a specific anaerobic field test for aerobic gymnastics. In Earnest, C.P. (Ed.). PLoS One, 10(4), e0123115.Public Library of Science (PLoS). doi: 10.1371/journal.pone.0123115.
- Alves, C.R.R., da Cunha, T.F., da Paixão, N.A., & Brum, P.C. (2015). Aerobic exercise training as therapy for cardiac and cancer cachexia. Life Sci, 125, 9-14.Elsevier. doi: 10.1016/j.lfs.2014.11.029.
- Abujabra Merege Filho, C.A., Rodrigues Alves, C.R., Sepulveda, C.A., Costa, A.D.S., Lancha Junior, A.H., & Gualano, B. (2014). INFLUENCE OF PHYSICAL EXERCISE ON COGNITION: AN UPDATE ON PHYSIOLOGICAL MECHANISMS. REVISTA BRASILEIRA DE MEDICINA DO ESPORTE, 20(3), 237-241.FapUNIFESP (SciELO). doi: 10.1590/1517-86922014200301930.
- Alves, C.R.R., Tessaro, V.H., Teixeira, L.A.C., Murakava, K., Roschel, H., Gualano, B., & Takito, M.Y. (2014). Influence of acute high-intensity aerobic interval exercise bout on selective attention and short-term memory tasks. Percept Mot Skills, 118(1), 63-72.SAGE Publications. doi: 10.2466/22.06.PMS.118k10w4.
- de Campos-Ferraz, P.L., Andrade, I., das Neves, W., Hangai, I., Alves, C.R.R., & Lancha, A.H. (2014). An overview of amines as nutritional supplements to counteract cancer cachexia. J Cachexia Sarcopenia Muscle, 5(2), 105-110.Wiley. doi: 10.1007/s13539-014-0138-x.
- Gualano, B., Macedo, A.R., Alves, C.R.R., Roschel, H., Benatti, F.B., Takayama, L., de Sá Pinto, A.L., Lima, F.R., & Pereira, R.M.R. (2014). Creatine supplementation and resistance training in vulnerable older women: a randomized double-blind placebo-controlled clinical trial. Exp Gerontol, 53, 7-15.Elsevier. doi: 10.1016/j.exger.2014.02.003.
- Alves, C.R.R., Campos, J.C., Moreira, J.B.N., Nobre, T.S., Sanches, I.C., de Moraes Forjaz, C.L., & Brum, P.C. (2013). Fisiologia do Exercício para alunos de graduação: uso de estratégias de ensino baseadas na metodologia dialética. Revista Brasileira de Educação Física e Esporte, 27(2), 289-296.FapUNIFESP (SciELO). doi: 10.1590/s1807-55092013000200012.
- Alves, C.R.R., Merege Filho, C.A.A., Benatti, F.B., Brucki, S., Pereira, R.M.R., de Sá Pinto, A.L., Lima, F.R., Roschel, H., & Gualano, B. (2013). Creatine supplementation associated or not with strength training upon emotional and cognitive measures in older women: a randomized double-blind study. In Blachier, F. (Ed.). PLoS One, 8(10), e76301.Public Library of Science (PLoS). doi: 10.1371/journal.pone.0076301.
- Alves, C.R.R., Santiago, B.M., Lima, F.R., Otaduy, M.C.G., Calich, A.L., Tritto, A.C.C., de Sá Pinto, A.L., Roschel, H., Leite, C.C., Benatti, F.B., Bonfá, E., & Gualano, B. (2013). Creatine supplementation in fibromyalgia: a randomized, double-blind, placebo-controlled trial. Arthritis Care Res (Hoboken), 65(9), 1449-1459.Wiley. doi: 10.1002/acr.22020.
- Alves, C.R., Gualano, B., Takao, P.P., Avakian, P., Fernandes, R.M., Morine, D., & Takito, M.Y. (2012). Effects of acute physical exercise on executive functions: a comparison between aerobic and strength exercise. J Sport Exerc Psychol, 34(4), 539-549.Human Kinetics. doi: 10.1123/jsep.34.4.539.
- Alves, C.R., Murai, I.H., Ramona, P., Nicastro, H., Bechara, L.R., Lancha, A.H., Brum, P.C., Irigoyen, M.C., & Gualano, B. (2012). No effect of creatine supplementation on oxidative stress and cardiovascular parameters in spontaneously hypertensive rats. J Int Soc Sports Nutr, 9(1), 13.Taylor & Francis. doi: 10.1186/1550-2783-9-13.
- Alves, C.R.R., Ferreira, J.C., de Siqueira-Filho, M.A., Carvalho, C.R., Lancha, A.H., & Gualano, B. (2012). Creatine-induced glucose uptake in type 2 diabetes: a role for AMPK-α?. Amino Acids, 43(4), 1803-1807.Springer Nature. doi: 10.1007/s00726-012-1246-6.
- Alves, C.R.R., Pasqua, L., Artioli, G.G., Roschel, H., Solis, M., Tobias, G., Klansener, C., Bertuzzi, R., Franchini, E., Lancha Junior, A.H., & Gualano, B. (2012). Anthropometric, physiological, performance, and nutritional profile of the Brazil National Canoe Polo Team. J Sports Sci, 30(3), 305-311.Taylor & Francis. doi: 10.1080/02640414.2011.638086.
- Roschel, H., Neves-Junior, M., Gualano, B., Barroso, R., Robles, C., de Sá Pinto, A.L., Fuller, R., & Lima, F.R. (2011). Familiarisation with lower limb strength testing in middle-aged women with osteoarthritis of the knee. Physiotherapy, 97(4), 350-353.Elsevier. doi: 10.1016/j.physio.2011.01.007.
- Fong, C., Alber, E., Gajjar, S., Das, S., Krishnan, V., Kalailingam, P., Roh, K., Alves, C., Yun, S., Fox, L., Ha, L., Stutzman, H., De Rossi, G., Kleinstiver, B., Maguire, C., Lindsay, M., Chung, D., & Musolino, P. (2025). Targeted Gene Correction Using AAV-Delivered Base Editing Improves Smooth Muscle Cell Function in the Eye of an ACTA2R179H MSMDS Mouse Model: A Promising Surrogate Endpoint For Future Clinical Trials. In MOLECULAR THERAPY, 33(4).
- Fong, C.E., Alber, E., Gajjar, S., Krishnan, V., Das, S., Roh, K., Alves, C., Yun, S., Fox, L., Ha, L., Stutzman, H., Kleinstiver, B., Maguire, C., Lindsay, M., Chung, D., & Musolino, P. (2024). Systemic AAV-Delivered Base Editing of ACTA2R179H Improves Pupillary Smooth Muscle Cell Contraction in Mice: A Promising Target Engagement Biomarker to Predict Clinical Efficacy. In MOLECULAR THERAPY, 32(4), (pp. 795-796).
- Krishnan, V., Das, S., Alves, C., Cardenas, C.L., Imai, T., Hai, J., Gandhi, A., Kalailingam, P., Chung, D., Lindsay, M.E., Kleinstiver, B.P., & Musolino, P.L. (2024). Systemic AAV-Delivered Targeted Gene Editing Restores Cerebrovascular Disease and Prevents Associated Neurodegeneration in ACTA2 R179H-Related Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS). In MOLECULAR THERAPY, 32(4), (pp. 313-314).
- Metovic, J., Li, Y., Alves, C.R.R., Rufino-Ramos, D., Ha, L.L., Golovanov, N., Gong, Y., Kleinstiver, B.P., & Eichler, F. (2024). Base Editor-Mediated Correction of a CSF1R Mutation That Causes ALSP. In MOLECULAR THERAPY, 32(4), (p. 344).
- Alves, C.R.R., Leillani, H.L., Yaworski, R., Lazzarotto, C.R., Christie, K., Reilly, A., Beauvais, A., Doll, R.M., de la Cruz, D., Maguire, C.A., Swoboda, K.J., Tsai, S.Q., Kothary, R., & Kleinstiver, B.P. (2023). PAMless Base Editors as a Genetic Treatment for Spinal Muscular Atrophy. In MOLECULAR THERAPY, 31(4), (p. 259).
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