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Amar Gajjar, MD
Amar Gajjar, MD

Amar Gajjar, MD

Member, St. Jude Faculty

  • Chair, Department of Pediatric Medicine
  • Co-Chair, Department of Oncology
  • Scott and Tracie Hamilton Endowed Chair in Brain Tumor Research




MD – Grant Medical College, J.J. Group of Hospitals, Bombay, India

Clinical Interests

My current clinical research interests focus on innovative protocols for the treatment of childhood brain tumors, with a focus on medulloblastomas, PNET and Rhabdoid tumors (ATRT). We just completed a large study (SJMB 96) in which 162 patients were treated. We used a strategy of risk-adapted radiation therapy followed by four cycles of high-dose chemotherapy with stem cell support. We achieved very good results with this approach. Our current study (SJMB 03) opened in September 2003. In this study we are doing extensive biological studies on the tumor tissue so that we can better understand what makes a normal brain cell turn into a tumor cell. We anticipate that this information will help us find new therapies to treat high-risk tumors and allow us to safely reduce therapy in patients that we determine are low-risk patients.

In addition to the biological studies on our SJMB 03 protocol, for the first time ever we are using a neurocognitive rehabilitation strategy to help children preserve their reading skills. This is the first study to prospectively ascertain if such intervention does indeed help children. Additionally, we are doing extensive imaging studies and detailed neuropsychological examinations to better understand the nature of the cognitive deficits so we can devise better strategies to help our children.

Research Interests

I am working with investigators in basic science laboratories to develop new chemotherapeutic agents for the treatment of brain tumors. Collaborative studies seek to unravel the mechanisms by which key anticancer drugs work so we can use them more efficiently.

Selected Publications

Northcott, PA, Buchhalter I, Morrissy AS, Hovestadt V, Groebner S, Ehrenberger T, Segura-Wang M, Zichner T, Weischenfeldt J, Schumacher S, Phillips AH, Kleinheinz K, Waszak SM, Erkek S, Jones DTW, Worst BC, Kool M, Zapatka M, Jäger N, Chavez L, Hutter B, Bieg M, Paramasivam N, Heinold M, Gu Z, Warnatz H-J, Risch T, Amstislavskiy V, Weber U, Wolf S, Robinson GW, Zhou X, Wu G, Finkelstein D, Cavalli F MG, Luu B, Ramaswamy V, Wu X, Koster J, Ryzhova M, Cho Y-J, Pomeroy S, Herold-Mende C, Schuhmann M, Ebinger M, Liau LM, Mora J, McLendon RE, Jabado N, Kumabe T, Witt O, Milde T, Von Deimling A, Capper D, Korshunov A, Yaspo M-L, Kriwacki R, Gajjar A, Zhang J, Beroukhim R, Fraenkel E, Korbel JO, Brors B, Schlesner M, Eils R, Marra MA, Pfister SM, Taylor MD, Lichter P. Comprehensive molecular profiling identifies novel actionable drivers and subtypes underlying medulloblastoma. Nature 2017 (in press).

Parikh KA, Venable G, Orr BA, Choudhri AF, Boop FA, Gajjar A, Klimo Jr. P. Pediatric Pineoblastoma. Journal of Neurosurgery Pediatrics 2017 (in press).

Glass JO, Ogg RJ, Hyun JW, Harreld JH, Li Y, Gajjar A, Reddick WE. Disrupted development and integrity of frontal white matter in patients treated for pediatric posterior fossa tumors. Neuro Oncology 2017 (in press).

Pajtler KW, Mack SC, Ramaswamy V, Smith CA, Witt H, Smith A, Hansford JR, von Hoff K, Wright KD, Hwang E, Frappaz D, Kanemura Y, Massimino M, Faure-Conter C, Modena P, Tabori U, Warren KE, Holland EC, Ichimura K, Giangaspero F, Castel D, von Deimling A, Kool M, Dirks PB, Grundy RG, Foreman NK, Gajjar A, Korshunov A, Finlay J, Gilbertson RJ, Ellison DW, Aldape KD, Merchant TE, Bouffet E, Pfister SM, Taylor MD. The current consensus on the clinical management of intracranial ependymoma and its distinct molecular variants. Acta Neuropathol 133(1):5-12, 2017. PMCID: PMC5209502.

Hoehn ME, Calderwood J, O'Donnell T, Armstrong GT, Gajjar A. Children with dorsal midbrain syndrome as a result of pineal tumors. J AAPOS Feb;21(1):34-38, 2017. PMID: 28069468

Sparrow J, Zhu L, Gajjar A, Mandrell BN, Ness KK. Constraint-Induced Movement Therapy for Children With Brain Tumors. Pediatr Phys Ther 29(1):55-61, 2017. PMCID: PMC5172401

Broniscer A, Hwang S, Chamdine O, Lin T, Pounds S, Onar-Thomas A, Chi L, Shurtleff S, Allen S, Gajjar A, Northcott P, Orr BA. Bithalamic gliomas are molecularly distinct from their unilateral high-grade counterparts. Acta Neuropathologica Communications. Brain Pathol Dec 28, 2016. [Epub ahead of print] PMID: 28032389

Torchia J, Golbourn B, Feng S, Ching Ho K, Sin-Chan P, Vasilijevic A, Norman JD, Guilhamon P, Garzia L, Agamez NR, Lu M, Chan TS Picard D, deAntonellis P, Quang DAK, Planello AC, Zeller C, Barsyte-Lovejoy D, Lafay-Cousin L, Letourneau L, Bourgey M, Yu M, Gendoo DMA, Dzamba M, Barszczyk M, Medina T, Riemenschneider AN, Morrisy AS, Ra, YS, Ramaswamy V, Remke M, Pimentel J, Chan JA, Somers GR, Faria CC, Roque L, Fouladi Mm Hoffman LM, Moore AS, Wang Yi,, Choi SA, Hansford JR, Catchpoole D, Birks DK, Foreman N, Strother D, Klekner A, Bognar L, Garami M, Hauser P, Hortobagyi T, Wilson B,  Hukin J, Carret AS, Van Meter TE, Hwang E, Gajjar A, Chiou SH, Nakamura H, Toledano H, Fried I, Fults D, Wataya T, Fryer C, Eisenstat DD, Scheineman K, Fleming AJ, Johnston D, Michaud J, Zelcer S, Hammond R, Afzal S, Ramsay DA, Sirachainan N, Hongeng S, Larbcharoensub N, Grundy RG, Lulla RR, Fangusaro JR, Phillips J, Majewski J, Montpetit A, Bourque G, Bader GD, Reddy AT, Gillespie YG, Warmuth-Metz M, Rutkowski S, Tabori U, Lupien M, Brundo M, Schuller, Pietsch T, Judkins AR, Hawkins CE, Bouffet E, Kim SK, Dirks P, Taylor MD, Epstein AE, Arrowsmith CH, De Carvalho DD, Rutka JT, Jabado N, Huang A. Integrated (epi)-Genomic Analysis Identify Subgroup-Specific Therapeutic Targets in CNS Rhabdoid Tumors. Cancer Cell 30(6):891-908, 2016. PMID: 27960086

Esbenshade AJ, Kocak M, Hershon L, Rousseau P, Decarie J-C, Shaw S, Burger P, Friedman HS, Gajjar A, Moghrabi A. A phase II feasibility study of oral etoposide given concurrently with radiotherapy followed by dose intensive adjuvant chemotherapy for children with newly diagnosed high-risk medulloblastoma; COG protocol POG 9631, A report from the Children’s Oncology Group. Pediatr Blood Cancer Dec 21, 2016. [Epub ahead of print] PMID: 28000417

Roberts M, Selvo N, Roberts J, Daryani V, Owens T, Hardstead E,  Gajjar A, Stewart C. Determination of Methotrexate, 7-Hydroxymethotrexate, and 2,4-Diamino-N10-methylpteroic Acid by LC-MS/MS in Plasma and Cerebrospinal Fluid and Application in a Pharmacokinetic Analysis of High-Dose Methotrexate. Journal of Liquid Chromatography & Related Technologies 2017 (in press).

Packer RJ, Pfister S, Bouffet E, Avery R, Bandopadhayay P, Bornhorst M, Bowers D, Ellison D, Fangusaro J, Foreman N, Fouladi M, Gajjar A, Haas-Kogan D, Hawkins C, Ho C-Y, Hwang E, Jabado N, Kilburn LB, Lassaletta A, Ligon KL, Massimino M, Meeteren S, Sabine M, Nicolaides T, Giorgio P, Tabori U, Vezina G, Warren K, Wit O, Yuan Zhu, David T Jones, Mark Kieran. Pediatric Low-Grade gliomas: Implications of the Biologic Era. Neuro Oncol Sep 28, 2016. [Epub ahead of print] Review. PMID: 27683733

Chemaitilly W, Armstrong GT, Gajjar A, Hudson MM. Hypothalamic-Pituitary Axis Dysfunction in Survivors of Childhood Central Nervous System Tumors: The Importance of Systematic Follow-up and Early Endocrine Consultation. Journal of Clinical Oncology 34:364315-4319, 2016. PMID: 27998231

Sadighi ZS, Zabrowski J, Broniscer A, Gajjar A, Boop FA, Khan R. Clinical characteristics and long-term outcome in movement disorder in childhood thalamic tumors. Pediatr Neurol 65:71-77, 2016. PMID: 27773422

Daryani VM, Patel YT, Tagen M, Turner DC, Carcaboso AM, Atkinson JM, Gajjar A, Gilbertson RJ, Wright KD, Stewart CF. Translational pharmacokinetic-pharmacodynamic modeling and simulation: Optimizing 5-fluorouracil dosing in children with pediatric ependymoma. CPT Pharmacometrics Syst Parmacol 5(4):211-221, 2016. PMID: 27109549

Mandrell BN, Baker JN, Levine D, Gattuso J, West N,Skyes A, Gajjar A, Broniscer A.  Patients with no chance for cure: Patient and parent proxy reported quality of life and the impact on the parental physical and mental health during the treatment trajectory. J Neurooncol 129(2):373-81, 2016. PMID: 27344555

Ramaswamy V, Hielscher T, Mack SC, Lassaletta A, Lin T, Pajtler KW, Jones DT, Luu B, Cavalli FM, Aldape K, Remke M, Mynarek M, Rutkowski S, Gururangan S, McLendon RE, Lipp ES, Dunham C, Hukin J, Eisenstat DD, Fulton D, van Landeghem FK, Santi M, van Veelen ML, Van Meir EG, Osuka S, Fan X, Muraszko KM, Tirapelli DP, Oba-Shinjo SM, Marie SK, Carlotti CG, Lee JY, Rao AA, Giannini C, Faria CC, Nunes S, Mora J, Hamilton RL, Hauser P, Jabado N, Petrecca K, Jung S, Massimi L, Zollo M, Cinalli G, Bognár L, Klekner A, Hortobágyi T, Leary S, Ermoian RP, Olson JM, Leonard JR, Gardner C, Grajkowska WA, Chambless LB, Cain J, Eberhart CG, Ahsan S, Massimino M, Giangaspero F, Buttarelli FR, Packer RJ, Emery L, Yong WH, Soto H, Liau LM, Everson R, Grossbach A, Shalaby T, Grotzer M, Karajannis MA, Zagzag D, Wheeler H, von Hoff K, Alonso MM, Tuñon T, Schüller U, Zitterbart K, Sterba J, Chan JA, Guzman M, Elbabaa SK, Colman H, Dhall G, Fisher PG, Fouladi M, Gajjar A, Goldman S, Hwang E, Kool M, Ladha H, Vera-Bolanos E, Wani K, Lieberman F, Mikkelsen T, Omuro AM, Pollack IF, Prados M, Robins HI, Soffietti R, Wu J, Metellus P, Tabori U, Bartels U, Bouffet E, Hawkins CE, Rutka JT, Dirks P, Pfister SM, Merchant TE, Gilbert MR, Armstrong TS, Korshunov A, Ellison DW, Taylor MD. Therapeutic Impact of Cytoreductive Surgery and Irradiation of Posterior Fossa Ependymoma in the Molecular Era: A Retrospective Multicohort Analysis. J Clin Oncol 34(21):2468-77 2016.PMID: 27269943

Roberts JK, Birg AV, Lin T, Daryani VM, Panetta JC, Broniscer A, Robinson GW, Gajjar AJ, Stewart CF. Population pharmacokinetics of oral topotecan in infants and very young children with brain tumors demonstrates a role of ABCG2 rs4148157 on the absorption rate constant. Drug Metab Dispos 44(7):1116-1122, 2016. PMCID: PMC4931885

Ramaswamy V, Remke M, Bouffet E, Bailey S, Clifford SC, Doz F, Kool M, Dufour C, Vassal G, Milde T, Witt O, von Hoff K, Pietsch T, Northcott PA, Gajjar A, Robinson GW, Padovani L, Andre N, Massimino M, Pizer B, Packer R, Rutkowski S, Pfister SM, Taylor MD, Pomeroy SL. Risk stratification of childhood medulloblastoma in the molecular era: the current consensus. Acta Neuropathol 131(6):821-831, 2016. PMCID: PMC4867119

Sturm D, Orr BA, Toprak UH, Hovestadt V, Jones DT, Capper D, Sill M, Buchhalter I, Northcott PA, Leis I, Ryzhova M, Koelsche C, Pfaff E, Allen SJ, Balasubramanian G, Worst BC, Pajtler KW, Brabetz S, Johann PD, Sahm F, Reimand J, Mackay A, Carvalho DM, Remke M, Phillips JJ, Perry A, Cowdrey C, Drissi R, Fouladi M, Giangaspero F, Łastowska M, Grajkowska W, Scheurlen W, Pietsch T, Hagel C, Gojo J, Lötsch D, Berger W, Slavc I, Haberler C, Jouvet A, Holm S, Hofer S, Prinz M, Keohane C, Fried I, Mawrin C, Scheie D, Mobley BC, Schniederjan MJ, Santi M, Buccoliero AM, Dahiya S, Kramm CM, von Bueren AO, von Hoff K, Rutkowski S, Herold-Mende C, Frühwald MC, Milde T, Hasselblatt M, Wesseling P, Rößler J, Schüller U, Ebinger M, Schittenhelm J, Frank S, Grobholz R, Vajtai I, Hans V, Schneppenheim R, Zitterbart K, Collins VP, Aronica E, Varlet P, Puget S, Dufour C, Grill J, Figarella-Branger D, Wolter M, Schuhmann MU, Shalaby T, Grotzer M, van Meter T, Monoranu CM, Felsberg J, Reifenberger G, Snuderl M, Forrester LA, Koster J, Versteeg R, Volckmann R, van Sluis P, Wolf S, Mikkelsen T, Gajjar A, Aldape K, Moore AS, Taylor MD, Jones C, Jabado N, Karajannis MA, Eils R, Schlesner M, Lichter P, von Deimling A, Pfister SM, Ellison DW, Korshunov A, Kool M. New brain tumor entities emerge from molecular classification of CNS-PNETs. Cell 164(5):1060-1072, 2016. PMCID: PMC5139621

Johann PD, Erkek S, Zapatka M, Kerl K, Buchhalter I, Hovestadt V, Jones DT, Sturm D, Hermann C, Segura Wang M, Korshunov A, Rhyzova M, Gröbner S, Brabetz S, Chavez L, Bens S, Gröschel S, Kratochwil F, Wittmann A, Sieber L, Geörg C, Wolf S, Beck K, Oyen F, Capper D, van Sluis P, Volckmann R, Koster J, Versteeg R, von Deimling A, Milde T, Witt O, Kulozik AE, Ebinger M, Shalaby T, Grotzer M, Sumerauer D, Zamecnik J, Mora J, Jabado N, Taylor MD, Huang A, Aronica E, Bertoni A, Radlwimmer B, Pietsch T, Schüller U, Schneppenheim R, Northcott PA, Korbel JO, Siebert R, Frühwald MC, Lichter P, Eils R, Gajjar A, Hasselblatt M, Pfister SM, Kool M. Atypical teratoid/rhabdoid tumors are comprised of three epigenetic subgroups with distinct enhancer landscapes. Cancer Cell 29:379-393, 2016. PMID: 26923874  

Broniscer A, Chamdine O, Hwang S, Lin T, Pounds, S, Onar-Thomas A, Shurtleff S, Gajjar A, Northcott PA, Orr BA. Gliomatosis cerebri in children shares molecular characteristics with other pediatric gliomas. Acta Neuropathol 131(2):299-307, 2016. PMCID: PMC4886851

Pui CH, Pappo A, Gajjar A, Downing JD. Redefining “rare” in pediatric cancers. Lancet Oncol 17(2):138-139, 2016. PMID: 26868337 [PubMed - in process]

Brinkman TM, Krasin MJ, Liu W, Armstrong GT, Ojha RP, Sadighi ZS, Gupta P, Kimberg C, Srivastava D, Merchant TE, Gajjar A, Robison LL, Hudson MH, Krull KR. Long-term neurocognitive functioning and social attainment in adult survivors of pediatric CNS tumors: Results from the St. Jude Lifetime Cohort Study. J Clin Oncol 34(12):1358-1367, 2016. PMCID:  PMC4933131

Khan RB, Sadighi ZS, Zabrowski J, Gajjar A, Jeha S. Imaging patterns and outcome of posterior reversible encephalopathy syndrome during childhood cancer treatment. Pediatr Blood Cancer 63(3):523-526, 2016. PMCID: PMC4724342

Morfouace M, Nimmervoll B, Boulos N, Patel YT, Shelat A, Freeman III BB, Robinson G, Wright K, Gajjar A, Stewart CF, Gilbertson R, Roussel MF. Preclinical studies of 5-fluoro-2’-deoxycytidine and tetrahydrouridine in pediatric brain tumors. J Neurooncol 126(2):225-234, 2016. PMCID: PMC4718940

Chemaitilly W, Merchant TE, Li Z, Barnes N, Armstrong GT, Ness KK, Pui C-H, Kun LE, Robison LL, Hudson MH, Sklar CA, Gajjar A. Central precocious puberty following pediatric cancers and brain tumors: Presentation and long-term outcomes. Clin Endocrinol (Oxf) 84(3):361-371, 2016. PMCID: PMC4755813

Chamdine O, Broniscer A, Wu S, Gajjar A, Qaddoumi I. Metastatic low-grade gliomas in children: 20 years' experience at St. Jude Children's Research Hospital. Pediatr Blood Cancer 63(1):62-70, 2016. PMCID: PMC5122937

Zou P, Conklin HM, Scoggins MA, Li Y, Jones MM, Palmer SL, Gajjar A, Ogg RJ. Functional MRI in medulloblastoma survivors supports prophylactic reading intervention during tumor treatment. Brain Imaging Behav 10(1):258-271, 2016. PMCID: PMC4644520

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Bratton ML, Hoehn ME, Morris B 3rd, Merchant TE, Gajjar A, Patel R, Kerr NC. Residual strabismus in children following improvement of cranial nerve palsies affecting ocular ductions. Am Orthopt J 65:87-93, 2015. PMID: 26564932 [PubMed - in process]

Brinkman TM, Bass JK, Li Z, Ness KK, Gajjar A, Pappo AS, Armstrong GT, Merchant TE, Srivastava DK, Robison LL, Hudson MM, Gurney JG. Treatment-induced hearing loss and adult social outcomes in survivors of childhood CNS and non-CNS solid tumors: Results from the St. Jude Lifetime Cohort Study. Cancer 121(22):4053-4061, 2015. PMCID: PMC4635051

Zhang J, Walsh MF, Wu G, Edmonson ME, Gruber TA, Easton J, Hedges D, Ma X, Zhou X, Yergeau DA, Wilkinson MR, Vadodaria B,  Chen C, McGee RB, Hines-Dowell S, Nuccio R,  Quinn E, Shurtleff SA,  Rusch M, Patel A, Becksfort JB, Wang S, Weaver MS, Ding L, Mardis ER, Wilson RK, Gajjar A, Ellison DW, Pappo AS, Pui CH, Nichols KE, Downing JR. Germline Mutations in Predisposition Genes in 1,120 Children with Cancer. N Engl J Med 373(24):2336-2346, 2015. PMCID: PMC4734119

Hoffman LM, Fouladi M, Olson J, Daryani VM, Stewart CF, Wetmore C, Kocak M, Onar-Thomas A, Wagner L, Gururangan S, Packer RJ, Blaney SM, Gajjar A, Kun LE, Boyett JM, Gilbertson RJ. Phase I trial of weekly MK-0752 in children with refractory central nervous system malignancies: a pediatric brain tumor consortium study. Childs Nerv Syst 31(8):1283-1289, 2015. PMCID: PMC4681692

Wright KD, Daryani VM, Turner DC, Onar-Thomas A, Boulos N, Orr BA, Gilbertson RJ, Stewart CF, Gajjar A. Phase 1 study of 5-fluorouracil in children and young adults with recurrent ependymoma. Neuro Oncol 17(12):1620-1627, 2015. PMCID: PMC4633933

Chintagumpala M, Eckel S, Krailo M, Morris M, Adesina A, Packer R, Lau C, Gajjar A. A pilot study using carboplatin, vincristine, and temozolomide in children with progressive/symptomatic low-grade glioma: a Children's Oncology Group study. Neuro Oncol 17(8):1132-1138, 2015. PMCID: PMC4490875

Gajjar A, Bowers DC, Karajannis M, Leary S, Witt H, Gottardo N. Pediatric brain tumors – Innovative genomics transforming the diagnostic and clinical landscape. J Clin Oncol 33(27):2986-2998, 2015. PMCID: PMC4567701

Green DM, Merchant TE, Billups CA, Stokes DC, Broniscer A, Bartels U, Chintagumpala M, Hassall TE, Gururangan S, McCowage GB, Heath JA, Cohn RJ, Fisher MJ, Srinivasan A, Robinson GW Gajjar A. Pulmonary function after treatment for embryonal brain tumors on SJMB03 that included craniospinal irradiation. Int J Radiat Oncol Biol Phys 93(1):47-53, 2015. PMCID: PMC4629817

DeWire M, Fouladi M, Turner DC, Wetmore C, Hawkins C, Jacobs C, Yuan Y, Liu D, Goldman S, Fisher P, Rytting M, Bouffet E, Khakoo Y, Hwang EI, Foreman N, Stewart CF, Gilbert MR, Gilbertson RJ, Gajjar A. An open-label, two-stage, phase II study of bevacizumab and lapatinib in children with recurrent or refractory ependymoma: A collaborative ependymoma research network study (CERN). J Neurooncol 123(1):85-91, 2015. PMCID: PMC4992988

Robinson GW, Orr BA, Wu G, Gururangan S, Lin T, Qaddoumi I, Packer R, Goldman S, Prados M, Desjardins A, Chintagumpala M,  Tabeke N, Rusch M, Gupta P, Thrasher A, Allen SJ, Li S, Stewart CF, Fouladi M, Boyett JM, Curran T, Gilbertson RJ, Ellison DW, Gajjar A. Vismodegib shows efficacy against recurrent SHH subtype medulloblastoma: Results of two phase II Pediatric Brain Tumor Consortium Studies - PBTC-025B & PBTC-032. J Clin Oncol 33(24):2646-2654, 2015. PMCID: PMC4534527

Cohen BH, Geyer JR, Miller DC, Curran JG, Zhou T, Sposto R, Holmes E, Ingles S, Dunkel I, Packer RJ, Pollack IF, Gajjar A, Finlay J. Pilot study of intensive chemotherapy with peripheral hematopoietic cell support for children less than 3 years of age with malignant brain tumors, the CCG-99703 phase I/II study. A report from the Children's Oncology Group. Pediatr Neurol 53(1):31-46, 2015. PMCID: PMC5166616

Arnautovic A, Billups C, Broniscer A, Gajjar A, Boop FA, Qaddoumi I. Delayed diagnosis of childhood low-grade glioma: causes, consequences and potential solutions. Childs Nerv Syst 31(7):1067-1077, 2015. PMCID: PMC4496265

Wetmore C, Boyett J, Li S, Bendel A, Gajjar A, Orr B. Alisertib is active as single agent in recurrent atypical teratoid rhabdoid tumors in 4 children. Neuro Oncol 17(6):882-888, 2015. PMCID: PMC4483126

Wright KD, Sabin ND, Cheuk D, McNall-Knapp R, Shurtleff SA, Gajjar A, Broniscer A. Incidental diagnosis of diffuse intrinsic pontine glioma in children. Pediatr Blood Cancer 62(6):1081-1083, 2015. PMCID: PMC4405458

Xu H, Robinson GW, Huang J, Lim JY, Bass J, Broniscer A, Chintagumpala M, Bartels U, Gururangan S, Hassall T, Fisher MJ, Cohn RC, Zuo J, Onar-Thomas A, Gajjar A, Stewart CF, Yang JJ. Common variants in ACYP2 influence the susceptibility to cisplatin-induced hearing loss. Nat Genet 47(3):263-266, 2015. PMCID: PMC4358157

Last update: June 2017