TERÀPIA GÈNICA

Presentació treballs
2021/2022

Grup Cognoms i nom Tutor Data

MOLINER ROLDAN, Manuel MIQUEL GARCIA

1
27/04/22
JIMÉNEZ JORDÁN, Jesús Telf: 4192
E-mail: Miquel.Garcia@uab.cat
Article:
In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice. Nature. 2021 Jan;589(7843):608-
614. doi: 10.1038/s41586-020-03086-7. Epub 2021 Jan 6.

PINEDA MICOLA, Anna MIQUEL GARCIA

3
27/04/22
MÉRIDA PÉREZ, Andrea Telf: 4192
E-mail: Miquel.Garcia@uab.cat
Article:
VCN-01 disrupts pancreatic cancer stroma and exerts antitumor effects. J Immunother Cancer. 2021
Nov;9(11):e003254. doi: 10.1136/jitc-2021-003254.

ALBINYÀ PEDRÓS, Alba MIQUEL GARCIA

4
27/04/22
RUBIO SILVAN, Laura Telf: 4192
E-mail: Miquel.Garcia@uab.cat
Article:
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A. N Engl J Med. 2020 Jan 2;382(1):29-40.
doi: 10.1056/NEJMoa1908490.

GÓMEZ MARTÍNEZ, Rosario Almudena SARA MARCÓ

5
27/04/22
SÁNCHEZ SAURA, Olaya Telf: 4181
E-mail: Sara.Marco@uab.cat
Article:
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse
models of infantile parkinsonism. Sci Transl Med. 2021 May 19;13(594):eaaw1564. doi:
10.1126/scitranslmed.aaw1564.

AVALLE RODRÍGUEZ, Silvia SARA MARCÓ

6
29/04/22
URIO GARMENDIA, Garazi Telf: 4181
E-mail: Sara.Marco@uab.cat
Article:
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration. Mol Ther
Methods Clin Dev. 2022 Jan 10;24:210-221. doi: 10.1016/j.omtm.2022.01.002. eCollection 2022 Mar 10.

RODRIGO HERNÁNDEZ, Silvia SARA MARCÓ

7
29/04/22
RAMIREZ GOMEZ, David Telf: 4181
E-mail: Sara.Marco@uab.cat
Article:
Regulated control of gene therapies by drug-induced splicing. Nature. 2021 Aug;596(7871):291-295. doi:
10.1038/s41586-021-03770-2. Epub 2021 Jul 28.
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OLMOS GIMÉNEZ, Carlota SARA MARCÓ

8
29/04/22
GARCIA TERCERO, Laura Telf: 4181
E-mail: Sara.Marco@uab.cat
Article.
Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh síndrome.
Mol Ther Methods Clin Dev. 2021 Sep 7;23:158-168. doi: 10.1016/j.omtm.2021.09.001. eCollection 2021 Dec 10.

CASANOVAS SANGLAS, Guillem SARA MARCÓ

9
29/04/22
GÓRRIZ VERGÉS, Albert Telf: 4181
E-mail: Sara.Marco@uab.cat
Article:
Lentivirus-mediated gene therapy for Fabry disease. Nat Commun. 2021 Feb 25;12(1):1178. doi: 10.1038/s41467-
021-21371-5.

MARTÍN MIRAS, María del Carmen ALBERT RIBERA

10
29/04/22
JIMÉNEZ NAVARRO, Ainara Telf: 4184
E-mail: Albert.Ribera@uab.cat
ROMAGUERA PALOMÉ, Aina
Articles:
Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders. Nat
Commun. 2019 Jan 22;10(1):236. doi: 10.1038/s41467-018-08264-w.
Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice. Mol Ther. 2021
Mar 3;29(3):973-988. doi: 10.1016/j.ymthe.2020.11.016. Epub 2020 Nov 17.

BENEYTO CATALÁN, Claudia ALBERT RIBERA

11
02/05/22
PERLAZA MENESES, Danna Alejandra Telf: 4184
E-mail: Albert.Ribera@uab.cat
Article:
Brain-wide Cas9-mediated cleavage of a gene causing familial Alzheimer's disease alleviates amyloid-related
pathologies in mice. Nat Biomed Eng. 2022 Feb;6(2):168-180. doi: 10.1038/s41551-021-00759-0. Epub 2021 Jul 26.

RAMIREZ MARTINEZ, Nerea ALBERT RIBERA

12
02/05/22
PRIETO MUÑOZ, Núria Telf: 4184
E-mail: Albert.Ribera@uab.cat
Article:
Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral
abnormalities and EEG activity. Mol Ther Methods Clin Dev. 2021 Jul 16;22:196-209. doi:
10.1016/j.omtm.2021.06.013. eCollection 2021 Sep 10.

ALVAREZ BEGUÉ, Mar ALBERT RIBERA

13
02/05/22
PÉREZ LÓPEZ, Marta Telf: 4184
E-mail: Albert.Ribera@uab.cat

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PUIG GARCÍA, Laura ALBERT RIBERA

14
02/05/22
REDONDO MORENO, Sofia Telf: 4184
E-mail: Albert.Ribera@uab.cat
Article:
A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced
liver cells. Nat Biotechnol. 2021 Jan;39(1):47-55. doi: 10.1038/s41587-020-0741-7. Epub 2020 Nov 16.

JIMENO BRAVO, Inés VICTOR SANCHEZ

15
02/05/22
PARICIO NAVAS, María Telf: 4184
E-mail: Victor.Sanchez@uab.cat
Article:
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of
hemophilia A. Mol Ther Methods Clin Dev. 2021 Nov 24;24:20-29. doi: 10.1016/j.omtm.2021.11.005. eCollection 2022
Mar 10.

GARCÍA MONLEÓN, Beatriz VICTOR SANCHEZ

16
04/05/22
GUASCH CAPELLA, Nora Telf: 4184
E-mail: Victor.Sanchez@uab.cat
Article:
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1. Gene Ther. 2021 Feb 4. doi:
10.1038/s41434-021-00231-3. Online ahead of print.

SANROMÀ MANI, Marta VICTOR SANCHEZ

17
04/05/22
POSAS SOLANES, Guillem Telf: 4184
E-mail: Victor.Sanchez@uab.cat
Article:
AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria. Mol Ther Methods Clin Dev. 2019 Dec
24;17:234-245. doi: 10.1016/j.omtm.2019.12.004. eCollection 2020 Jun 12.

CLOTET I REY, Laura VICTOR SANCHEZ

18
04/05/22
GINÉS PÉREZ, Paula Telf: 4184
E-mail: Victor.Sanchez@uab.cat
Article:
AAV-mediated in vivo CAR gene therapy for targeting human T-cell leukemia. Blood Cancer J. 2021 Jun
23;11(6):119. doi: 10.1038/s41408-021-00508-1.

IGNES ROMEU, Aitana VICTOR SANCHEZ

19
04/05/22
TUBAU CAMPUZANO, Ot Telf: 4184
E-mail: Victor.Sanchez@uab.cat
Article:
BCLX L gene therapy moderates neuropathology in the DBA/2J mouse model of inherited glaucoma. Cell Death
Dis. 2021 Aug 10;12(8):781. doi: 10.1038/s41419-021-04068-x.

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BALCELLS DE PABLO, Biel XAVIER LEON

20
04/05/22
MUNNÉ SURROCA, Marina Telf: 4181
E-mail: xavier.leon@uab.cat
Article:
Genome editing of mutant KRAS through supramolecular polymer-mediated delivery of Cas9
ribonucleoprotein for colorectal cancer therapy. J Control Release. 2020 Jun 10;322:236-247. doi:
10.1016/j.jconrel.2020.03.015. Epub 2020 Mar 10.

VIÑALS CERVERA, Ivan XAVIER LEON

21
06/05/22
VALENCIA SALCEDO, Nikolas Telf: 4181
E-mail: xavier.leon@uab.cat
Article:
Targeting the Apoa1 locus for liver-directed gene therapy. Mol Ther Methods Clin Dev. 2021 Apr 24;21:656-669.
doi: 10.1016/j.omtm.2021.04.011. eCollection 2021 Jun 11.

FERNANDEZ DOURADO, Juan XAVIER LEON

22
06/05/22
ARENAS FARHANE, Sara Telf: 4181
E-mail: xavier.leon@uab.cat
Article:
Virally programmed extracellular vesicles sensitize cancer cells to oncolytic virus and small molecule therapy.
Nat Commun. 2022 Apr 7;13(1):1898. doi: 10.1038/s41467-022-29526-8.

GÓMEZ LORES, Oriol XAVIER LEON

23
06/05/22
PARRAMON CODINA, Roger Telf: 4181
E-mail: xavier.leon@uab.cat
JAURRIETA CASTANY, Iker
Article:
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021 Apr 1;384(13):1216-1226.
doi: 10.1056/NEJMoa2021712.
Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled
Randomized Clinical Trial. Clin J Am Soc Nephrol. 2021 Jul;16(7):1025-1036. doi: 10.2215/CJN.14730920. Epub
2021 May 13.

COSTALS GELABERT, Maria XAVIER LEON

24
06/05/22
FERNÁNDEZ SOBRAL, Nicolás Telf: 4181
E-mail: xavier.leon@uab.cat
Article:
Gene editing and elimination of latent herpes simplex virus in vivo. Nat Commun. 2020 Aug 18;11(1):4148. doi:
10.1038/s41467-020-17936-5.

GRAJEA GONZ LEZ, Estel MERITXELL MORRÓ

25
06/05/22
SAULEDA SAULEDA, Judith Telf: 4185
E-mail: Meritxell.Morro@uab.cat
Article:
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model. Gene Ther. 2022 Feb
1. doi: 10.1038/s41434-022-00313-w. Online ahead of print.

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MONTES RUIZ, Lídia MERITXELL MORRÓ

26
09/05/22
ZARUMA DELGADO, Maria José Telf: 4185
E-mail: Meritxell.Morro@uab.cat
PORTA TOVAR, Guillem
Article:
MGMT genomic rearrangements contribute to chemotherapy resistance in gliomas. Nat Commun. 2020 Aug
4;11(1):3883. doi: 10.1038/s41467-020-17717-0.
Gene Therapy for Drug-Resistant Glioblastoma via Lipid-Polymer Hybrid Nanoparticles Combined with
Focused Ultrasound. Int J Nanomedicine. 2021 Jan 8;16:185-199. doi: 10.2147/IJN.S286221. eCollection 2021.

LERTXUNDI OLAZABAL, Nagore MERITXELL MORRÓ

27
09/05/22
WYATT DOMERCQ, Irati Telf: 4185
E-mail: Meritxell.Morro@uab.cat
Article:
Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA. Nature. 2020
Nov;587(7833):281-284. doi: 10.1038/s41586-020-2835-2. Epub 2020 Oct 21.

MARTÍNEZ LACALLE, Anna MERITXELL MORRÓ

28
09/05/22
FAÚNDEZ VIDIELLA, Clàudia Telf: 4185
E-mail: Meritxell.Morro@uab.cat
ESTRADA BES, Berta
Articles:
Generation of pure monocultures of human microglia-like cells from induced pluripotent stem cells. Stem Cell
Res. 2020 Dec;49:102046. doi: 10.1016/j.scr.2020.102046. Epub 2020 Oct 14.
Alzheimer's Risk Gene TREM2 Determines Functional Properties of New Type of Human iPSC-Derived
Microglia. Front Immunol. 2021 Feb 3;11:617860. doi: 10.3389/fimmu.2020.617860. eCollection 2020.

MONTANÉGIL, Paula MERITXELL MORRÓ

29
09/05/22
PARÉS BORRELL, Teo Telf: 4185
E-mail: Meritxell.Morro@uab.cat
Article:
Mesenchymal Stem Cells Successfully Deliver Oncolytic Virotherapy to Diffuse Intrinsic Pontine Glioma. Clin
Cancer Res. 2021 Mar 15;27(6):1766-1777. doi: 10.1158/1078-0432.CCR-20-1499. Epub 2020 Dec 3.

VIÑES RODRÍGUEZ, Judith MARISA JAÉN

30
09/05/22
RUIZ HOLGUERA, Marina Telf: 4177
E-mail: marialuisa.jaen@uab.cat
RION I GUILLEMAT, Maria
Articles:
Non-viral delivery of chemically modified mRNA to the retina: Subretinal versus intravitreal administration. J
Control Release. 2019 Aug 10;307:315-330. doi: 10.1016/j.jconrel.2019.06.042. Epub 2019 Jun 29.
The obstacle course to the inner retina: Hyaluronic acid-coated lipoplexes cross the vitreous but fail to
overcome the inner limiting membrane. Eur J Pharm Biopharm. 2019 Aug;141:161-171. doi:
10.1016/j.ejpb.2019.05.023. Epub 2019 May 28.

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RAMOS SALA, Marc MARISA JAÉN

31
11/05/22
GÁLVEZ SANCHEZ, Aina Telf: 4177
E-mail: marialuisa.jaen@uab.cat
Article:
Neuroregenerative gene therapy to treat temporal lobe epilepsy in a rat model. Prog Neurobiol. 2022
Jan;208:102198. doi: 10.1016/j.pneurobio.2021.102198. Epub 2021 Nov 28.

GARCÍA GONZÁLEZ, Andrea MARISA JAÉN

32
11/05/22
GARCÍA RODRÍGUEZ, María Telf: 4177
E-mail: marialuisa.jaen@uab.cat
Article:
Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders. EMBO Mol Med . 2021 Apr
9;13(4):e13392. doi: 10.15252/emmm.202013392. Epub 2021 Feb 22.

JÁÑEZ SANTANA, Álvaro MARISA JAÉN

33
11/05/22
CARNICERO FUERTES, Alberto Telf: 4177
E-mail: marialuisa.jaen@uab.cat

BÁRCENAS LÓPEZ, Oriol MARISA JAÉN

34
11/05/22
BOURACHED SILVA, Nour Al Youssef Telf: 4177
E-mail: marialuisa.jaen@uab.cat
Article:
AAV gene therapy for Tay-Sachs disease. Nat Med. 2022 Feb;28(2):251-259. doi: 10.1038/s41591-021-01664-4.
Epub 2022 Feb 10.

GALLARDO URBÓN, María MARIA MOLAS

35
11/05/22
FEBRER RIERA, Maria Antònia Telf: 4181
E-mail: Maria.Molas@uab.cat
Article:
Intracellular prodrug gene therapy for cancer mediated by tumor cell suicide gene exosomes. Int J Cancer.
2021 Jan 1;148(1):128-139. doi: 10.1002/ijc.33188. Epub 2020 Aug 4.

FORTUNY GALINDO, Laia MARIA MOLAS

36
13/05/22
BOSCH JUAN, Marina Telf: 4181
E-mail: Maria.Molas@uab.cat
Article:
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 May
27;384(21):2002-2013. doi: 10.1056/NEJMoa2027675. Epub 2021 May 11.

ROMERO LIMONES, Darío Rafael MARIA MOLAS

37
13/05/22
SOTILLO SOTILLO, Paula Telf: 4181
E-mail: Maria.Molas@uab.cat
Article:
Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature. 2021 Jul;595(7866):295-
302. doi: 10.1038/s41586-021-03609-w. Epub 2021 Jun 2.

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VIDAL TEUTON, Jana Michelle MARIA MOLAS

38
13/05/22
VALLBONA ROMERO, Nuria Telf: 4181
E-mail: Maria.Molas@uab.cat
Article:
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced
by a mito-targeted mutant ND1 gene. Gene Ther . 2022 Apr 6. doi: 10.1038/s41434-022-00333-6. Online ahead of
print.

FARRÉ TARRATS, Laia MARIA MOLAS

39
13/05/22
DÍAZ CANO CAZORLA, Laura Telf: 4181
E-mail: Maria.Molas@uab.cat
Article:
Systemic AAV8-mediated delivery of a functional copy of muscle glycogen phosphorylase (Pygm) ameliorates
disease in a murine model of McArdle disease. Hum Mol Genet. 2020 Jan 1;29(1):20-30. doi: 10.1093/hmg/ddz214.

RODRIGUEZ REVERTE, Irina IGNASI GRASS

40
13/05/22
PEREA BLANCO, Luz María Telf: 4177
E-mail: Ignasi.Grass@uab.cat
Article:
CRISPR-Based Adenine Editors Correct Nonsense Mutations in a Cystic Fibrosis Organoid Biobank. Cell Stem
Cell. 2020 Apr 2;26(4):503-510.e7. doi: 10.1016/j.stem.2020.01.019. Epub 2020 Feb 20.

GABERNET GARRIGA, Maria IGNASI GRASS

41
16/05/22
FONSECA PAIVA, Bruna Sofia Telf: 4177
E-mail: Ignasi.Grass@uab.cat
DÍAZ VALLEJOS, Ethy Greace
Articles:
Scaffold-mediated CRISPR-Cas9 delivery system for acute myeloid leukemia therapy. Sci Adv. 2021 May
19;7(21):eabg3217. doi: 10.1126/sciadv.abg3217. Print 2021 May.
Codelivery of CRISPR-Cas9 and chlorin e6 for spatially controlled tumor-specific gene editing with synergistic
drug effects. Sci Adv. 2020 Jul 15;6(29):eabb4005. doi: 10.1126/sciadv.abb4005. eCollection 2020 Jul.

OBIOL SEBASTIÀ, Lídia IGNASI GRASS

43
16/05/22
URRUTIA HERRERA, Mireia Telf: 4177
E-mail: Ignasi.Grass@uab.cat
Article:
A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy
mutation. Mol Ther Methods Clin Dev. 2021 Jun 4;22:122-132. doi: 10.1016/j.omtm.2021.05.014. eCollection 2021
Sep 10.

JORDÁ PALLAROLAS, Mireia IGNASI GRASS

44
16/05/22
PACHA PILAR, Andrea Telf: 4177
E-mail: Ignasi.Grass@uab.cat
Article:
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked
hypophosphatemia. Sci Adv. 2021 Oct 29;7(44):eabj5018. doi: 10.1126/sciadv.abj5018. Epub 2021 Oct 27.

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FARRÉ BADIA, Judit CLAUDIA JAMBRINA

45
16/05/22
CAAMAÑO DEUS, Ana Telf: 4177
E-mail: Claudia.Jambrina@uab.cat
Article:
Highly efficient and tumor-selective nanoparticles for dual-targeted immunogene therapy against cancer. Sci
Adv. 2020 Jan 15;6(3):eaax5032. doi: 10.1126/sciadv.aax5032. eCollection 2020 Jan.

LLORENTE LLORENTE, Sofía VICTOR SACRISTAN

46
18/05/22
GARMENDIA DULFOVA, Vera Telf: 4177
E-mail: Victor.Sacristan@uab.cat

TRILLA BAJONA, Paula VICTOR SACRISTAN

47
18/05/22
GARCÍA FERNÁNDEZ, María Telf: 4177
E-mail: Victor.Sacristan@uab.cat
Article:
In vivo CRISPR/Cas9 targeting of fusion oncogenes for selective elimination of cancer cells. Nat Commun 2020
Oct 8;11(1):5060. doi: 10.1038/s41467-020-18875-x.

FRANQUESA SÁNCHEZ, Aleix GEMMA ELIAS

48
18/05/22
MONTE HOMS, Alex Telf: 4177
E-mail: Gemma.Elias@uab.cat
Article:
Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria síndrome. Nat Med. 2019
Mar;25(3):423-426. doi: 10.1038/s41591-018-0338-6. Epub 2019 Feb 18.

QUEROL JOVER, Judit GEMMA ELIAS

49
18/05/22
SITJÀ ROMERO, Gemma Telf: 4177
E-mail: Gemma.Elias@uab.cat
Article:
Preclinical development and phase 1 trial of a novel siRNA targeting lipoprotein(a). Nat Med. 2022 Jan;28(1):96-
103. doi: 10.1038/s41591-021-01634-w. Epub 2022 Jan 13.

GODOY FERNÁNDEZ, José Manuel GEMMA ELIAS

50
18/05/22
MARTÍNEZ GARCIA, Noemí Telf: 4177
E-mail: Gemma.Elias@uab.cat
Article:
CRISPR-Cas9 Mediated Exonic Disruption for HIV-1 Elimination. EBioMedicine. 2021 Nov;73:103678. doi:
10.1016/j.ebiom.2021.103678. Epub 2021 Nov 10.

ROMEI, Melissa GEMMA ELIAS

51
20/05/22
ESQUINAS LÓPEZ, Cristina Telf: 4177
E-mail: Gemma.Elias@uab.cat
RAMÍREZ GARCÍA, María

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PONS ESTÉVEZ, Sara GEMMA ELIAS

52
20/05/22
PUGA SÁNCHEZ, Miguel Telf: 4177
E-mail: Gemma.Elias@uab.cat
Article:
Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16
hearing loss. Sci Adv. 2021 Dec 17;7(51):eabi7629. doi: 10.1126/sciadv.abi7629. Epub 2021 Dec 15.

CARRASCO LOSANTOS, Mercè  MARTA PEREA

53
20/05/22
SÁNCHEZ CIRIA, Anna Telf: 4177
E-mail: Marta.Perea@uab.cat
Article:
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 Jan
21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5.

SUN, Chenyu MARTA PEREA

54
20/05/22
CALVO MEDINA, RocÍo Telf: 4177
E-mail: Marta.Perea@uab.cat
Article:
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis. N Engl J Med. 2021 Aug 5;385(6):493-502. doi:
10.1056/NEJMoa2107454. Epub 2021 Jun 26.

VIDAL BUESO, Alba MARTA PEREA

55
20/05/22
KESSELS SOLÉ, Marc Telf: 4177
E-mail: Marta.Perea@uab.cat
TORRES DOMÍNGUEZ, Laura
Articles:
CRISPR-Cas9-mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of
Leber congenital amaurosis. Sci Adv. 2019 Oct 30;5(10):eaax1210. doi: 10.1126/sciadv.aax1210. eCollection 2019
Oct.
Restoration of visual function in adult mice with an inherited retinal disease via adenine base editing. Nat
Biomed Eng. 2021 Feb;5(2):169-178. doi: 10.1038/s41551-020-00632-6. Epub 2020 Oct 19.

FORCADELL ORTIZ, Maria del Ara IGNASI GRASS

42
23/05/22
AIDOUN EL AMRANI, Salma Telf: 4177
E-mail: Ignasi.Grass@uab.cat
LÓPEZ CABEZÓN, Cristina

ASÍN I LÓPEZ, Laura MARTA PEREA

56
23/05/22
BENEDICTO MOLINA, Xavier Telf: 4177
E-mail: Marta.Perea@uab.cat
Article:
In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Nat Med.
2022 Mar 28. doi: 10.1038/s41591-022-01737-y. Online ahead of print.

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CARMONA JUÁREZ, Raquel MIQUEL GARCIA

2
23/05/22
FRANCO BORDES, Berta Telf: 4192
E-mail: Miquel.Garcia@uab.cat
GARCIA TÀPIAS, Pepa
Article:
Sleeping Beauty-engineered CAR T cells achieve antileukemic activity without severe toxicities. J Clin Invest.
2020 Nov 2;130(11):6021-6033. doi: 10.1172/JCI138473.
Bispecific CAR-T cells targeting both CD19 and CD22 for therapy of adults with relapsed or refractory B cell
acute lymphoblastic leukemia. J Hematol Oncol. 2020 Apr 3;13(1):30. doi: 10.1186/s13045-020-00856-8.

MURILLO HUART, Juan Carlos MARTA PEREA

57
23/05/22
MINGUELL CORTES, Gerard Telf: 4177
E-mail: Marta.Perea@uab.cat

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