9 research outputs found
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
- Author
- A Angelis
- A Annoni
- A Biffi
- A Cantore
- A Dhawan
- A Doenecke
- A Donsante
- A Ehrhardt
- A Ehrhardt
- A Fischer
- A Kiang
- A Mian
- A Mullard
- A Nowrouzi
- A Rosewell Shaw
- AA Rahim
- AC Nathwani
- AC Nathwani
- AC Nathwani
- AH Chang
- AK Zaiss
- AM Maguire
- AM Puumalainen
- AW Nienhuis
- B Crane
- B Thaci
- BD Brown
- BE Deverman
- C Booth
- C Bordignon
- C Kaeppel
- C Li
- C Mah
- C Summerford
- C Unzu
- CN Mattar
- CS Manno
- CS Manno
- D Duan
- D D’Avola
- D Majhen
- DA Muruve
- DD Koeberl
- DG Miller
- DM McCarty
- DM McCarty
- DP Rastall
- E Basner-Tschakarjan
- E Breous
- E Dolgin
- E Hastie
- EL Kuether
- EM Sokal
- EM Wilson
- EP Armstrong
- F Liu
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Schmitt
- F Vigant
- FD Ledley
- G Buchlis
- G Schiedner
- GC Pien
- GE Berry
- GJ McGarrity
- GP Gao
- GS Lipshutz
- H Azuma
- H Buning
- H Jiang
- H Jiang
- H Miyoshi
- HG Zhang
- HM Viecelli
- I Gil-Farina
- I Trapani
- Ian E. Alexander
- IM Verma
- J Haberle
- J Hoggatt
- J Lu
- J Pasi
- J Tang
- J Zhu
- JA Wolff
- JC Grieger
- JC Nault
- JE Rabinowitz
- JH McIntosh
- JL Andrews
- JM Coppoletta
- JM Wilson
- JN Lozier
- JR Counsell
- JS Powell
- Julien Baruteau
- JW Bainbridge
- K Benihoud
- K Jooss
- K Vercauteren
- KI Berns
- KM Flanigan
- L Apolonia
- L Cassiday
- L Couto
- L Lisowski
- L Sendra
- L Wang
- LF Earley
- LG Chicoine
- LM Belalcazar
- LM Kattenhorn
- LV Tse
- M Cavazzana-Calvo
- M Cerreto
- M Cooper
- M Elsabahy
- M Grossman
- M Grossman
- M Li
- M Naumer
- M Spada
- M Suzuki
- MA Kay
- MA Kay
- MA Kay
- MA Kotterman
- MK Chuah
- ML Brantly
- ML Hirsch
- N Bessis
- N Brunetti-Pierri
- N Brunetti-Pierri
- N Brunetti-Pierri
- N Brunetti-Pierri
- N Cartier
- N Junge
- N Morral
- N Touchot
- P Bell
- P Mayrhofer
- P Monahan
- P Piccolo
- Paul Gissen
- PE Monahan
- PS Reddy
- PT Clayton
- R Alba
- R Calcedo
- R Castello
- R Condiotti
- R Gebhardt
- R Gramignoli
- R Macaulay
- R Sarkar
- R Thwaite
- R Zufferey
- RD Hickey
- RJ Chandler
- RJ Chandler
- RJ Chandler
- RJ Yanez-Munoz
- RM Blaese
- RW Atchison
- RW Herzog
- S Boutin
- S Chira
- S Hacein-Bey-Abina
- S Hacein-Bey-Abina
- S Mukherjee
- S Nomura
- S Pillay
- SA Fahs
- SC Cunningham
- SC Cunningham
- SC Cunningham
- SE Raper
- SE Raper
- SE Raper
- SE Raper
- SH Orkin
- Simon N. Waddington
- SJ Nightingale
- SL Ginn
- SL Stephen
- SN Waddington
- SR Choudhury
- T Koo
- T Suwanmanee
- T Suzuki
- T Wirth
- T Yokoo
- TH Nguyen
- TR Flotte
- TR Flotte
- TR McKay
- V Louis Jeune
- V Monteilhet
- VR Arruda
- W Miesbach
- WM McCormack Jr
- WS Wold
- X Hou
- Y Chen
- Y Oishi
- Y Shi
- Y Yang
- Y Yang
- YS Tseng
- Publication venue
- Publication date
- 31/05/2017
- Field of study
Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vectors encoding the human factor IX cDNA. These trials demonstrate the potential of AAV technology to provide long-lasting clinical benefit in the treatment of monogenic liver disorders. Indeed, with more than ten ongoing or planned clinical trials for haemophilia A and B and dozens of trials planned for other inherited genetic/metabolic liver diseases, clinical translation is expanding rapidly. Gene therapy is likely to become an option for routine care of a subset of severe inherited genetic/metabolic liver diseases in the relatively near term. In this review, we aim to summarise the milestones in the development of gene therapy, present the different vector tools and their clinical applications for liver-directed gene therapy. AAV-derived vectors are emerging as the leading candidates for clinical translation of gene delivery to the liver. Therefore, we focus on clinical applications of AAV vectors in providing the most recent update on clinical outcomes of completed and ongoing gene therapy trials and comment on the current challenges that the field is facing for large-scale clinical translation. There is clearly an urgent need for more efficient therapies in many severe monogenic liver disorders, which will require careful risk-benefit analysis for each indication, especially in paediatrics
Acute Posttraumatic Cerebral Venous Sinus Thrombosis-Induced Malignant Increased Intracranial Pressure Treated with Endovascular Dural Sinus Thrombectomy and Stenting
- Author
- Publication venue
- 'Elsevier BV'
- Publication date
- Field of study
The Role of Energy in World Agriculture and Food Availability**Based on a talk first presented by the author at CENECA International Symposium on Energy, Paris, France, February 27–29, 1980.
- Author
- Publication venue
- 'Elsevier BV'
- Publication date
- 01/01/1984
- Field of study
Advances in gene therapy for hemophilia
- Author
- A Klukowska
- A Tiede
- AC Nathwani
- AC Nathwani
- B Mei
- B Nolan
- C Hu
- C Lyu
- C-Y Park
- C-Y Park
- C-Y Park
- C-Y Park
- CH Miao
- CS Manno
- D Melchiorre
- DA Roth
- EL Kuether
- F Rotblat
- H Matsui
- HH Roehl
- HZ Miao
- J Gitschier
- J Graw
- J Pang
- JA Dumont
- JJ Sung
- JM Staber
- JS Greengard
- JS Powell
- L Wang
- L Wang
- LA George
- M Franchini
- PM Mannucci
- PM Mannucci
- S Rangarajan
- S Shrestha
- SC Cunningham
- SC Gouw
- T Ohmori
- V Gabrovsky
- W Miesbach
- X Liu
- X Wang
- Y Guan
- Y Mizoguchi
- Y Wang
- Y Wu
- Publication venue
- 'Springer Science and Business Media LLC'
- Publication date
- Field of study
Progress toward inducing immunologic tolerance to factor VIII
- Author
- Aledort
- Alpan
- Andrés
- Anguela
- Annoni
- Arruda
- Auerswald
- Baker
- Borel
- Brackmann
- Bray
- Brown
- Burks
- Caram
- Carol H. Miao
- Cavazzana-Calvo
- Chai
- Check
- Chuah
- Clark
- Cromwell
- Dardikh
- David W. Scott
- De Groot
- DiMichele
- Ehrenforth
- El-Amine
- Ettinger
- Ettinger
- Getts
- Getts
- Gewirtz
- Gouw
- Hausl
- Hausl
- Herczenik
- Herzog
- Hoyer
- Hu
- Jaeckel
- James
- James
- Jiang
- Kang
- Kasper
- Kathleen P. Pratt
- Kempton
- Kuether
- Kwon
- Lei
- Lei
- Li
- Liu
- Liu
- Liu
- Liu
- LoDuca
- Mannucci
- Matsui
- Matsui
- Matzinger
- Miao
- Miao
- Miao
- Miao
- Miller
- Mingozzi
- Moayeri
- Moghimi
- Moise
- Nathwani
- Navarrete
- Nayak
- Nayak
- Ober
- Olafsen
- Pautard
- Peng
- Peng
- Peters
- Porada
- Porada
- Pratt
- Pratt
- Qadura
- Qian
- Qian
- Rath
- Rawle
- Reding
- Reding
- Reipert
- Rifle
- Ruhlman
- Sack
- Saint-Remy
- Sakaguchi
- Sarkar
- Scott
- Shapiro
- Skelley
- Skelton
- Skupsky
- Steinitz
- Su
- Su
- Sule
- Taylor
- Valentino
- van Haren
- Verma
- Wang
- Waters
- Weiner
- Wroblewska
- Wroblewska
- Yarovoi
- Ye
- Zhang
- Zhang
- Publication venue
- 'American Society of Hematology'
- Publication date
- Field of study
Das Stoffliche
- Author
- A Daniels
- A Daniels
- A Fischer
- A Gajdos
- A Henschel
- A Hock
- A Hock
- A Hock
- A Jores
- A Keys
- A Keys
- A Knudson
- A Lippmann
- A Löwy
- A Mirsky
- A Muralt
- A Palladin
- A PĂĽtter
- A Roche
- A Roche
- A Rossi
- A Scheunert
- A Scheunert
- A Scheunert
- A Scheunert
- A Schittenhelm
- A Szakáll
- A Szakáll
- A Szent Gyorgyi
- A Szent Gyorgyi
- A Szent Gyorgyi
- A Westerlund
- A Williamson
- AA Albanese
- AA Albanese
- AA Albanese
- AA Albanese
- AA Albanese
- AA Albanese
- AA Albanese
- AA Albanese
- AA Albanese
- AA Klose
- AA Klose
- AA Weech
- AA Weech
- AA Weech
- AD Holmes
- AD Welch
- AE Axelrod
- AE Hansen
- AE Houk
- AE Schaefer
- AE Schaefer
- AE Sobel
- AG Hogan
- AG Hogan
- AG Hogan
- AH Rafsky
- AH Smith
- AJ Antonow
- AJ Carlson
- AJ Carlson
- AJ Carlson
- AJ Glazebrook
- AJ Mueller
- ALS Cheng
- AM Yudkin
- AN Wick
- AR Kemmerer
- AR Mayer
- ARP Walker
- AT Fuller
- AW Donaldson
- AW Halverson
- AZ Hodson
- B Alexander
- B Ekman
- B Euler
- B Euler
- B Euler
- B Euler
- B Gustl
- B Jackson
- B Schmidt-Nielsen
- B Sjollema
- B Sure
- B Sure
- B Sure
- B Sure
- B SĂĽsskind
- B SĂĽsskind
- B SĂĽsskind
- BA Dor
- BB Migicovsky
- BC Gttha
- BD Westermann
- BD Westermann
- Benedict
- BH Ershoff
- BH Ershoff
- Bogert
- Borris
- Breitwieser
- Bruce
- BS Burke
- BS Burke
- BS Burke
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- BT Scheer
- BT Scheer
- Bunge
- BW Fairbanks
- C Boyd
- C Dumazert
- C Griebel
- C Heidelberger
- C Maase
- C Noorden
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- C Voit
- C Voit
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- CD Bauer
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- CF Asenjo
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- Councilon Foods u. Nutrition
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- D Melnick
- D Melnick
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- E Abderhalden
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- RD Eckhardt
- RD Eckhardt
- RD Williams
- RD Williams
- RE Spitzer
- RE Spitzer
- RF Loeb
- RH Chittenden
- RH Chittenden
- RH Common
- RH Lane
- RH Silber
- RH Silber
- RJ Block
- RJ Block
- RJ Evans
- RJ Williams
- RJ Williams
- RJ Williams
- RL Potter
- RM Campbell
- RM Johnson
- RM Leverton
- RM Leverton
- RM Tomarelli
- RO Neumann
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- RP Geyer
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- RR Sealock
- RR Sealock
- RR Sealock
- RR Williams
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- RT Conner
- RT Conner
- RW McKee
- RW Wissler
- S Ansbacher
- S Ansbacher
- S Black
- S Freeman
- S Granick
- S Kirkwood
- S Lepkovsky
- S Lepkovsky
- S Ochoa
- S Sherlock
- SA Harris
- SA Harris
- SA Localio
- SC Werner
- SC Werner
- SD Rubbo
- SE Dicker
- SE Smith
- SE Smith
- SG Smith
- SK Kon
- SL Thompsett
- SL Wilems
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- SS Zilva
- SW Klatzein
- SZ Levine
- T Hamada
- T Li
- T Moore
- T Pfeiffer
- T Pfeiffer
- TB Osborne
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- TB Osborne
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- TD Ltjckey
- TD Spies
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- TJ Haley
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- V Augur
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- VB Reader
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- VR Potter
- W Bachmann
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- W Heupke
- W Keil
- W Keil
- W Kempner
- W Lintzel
- W Menschick
- W Neuweiler
- W Schweizer
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- W Shive
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- WC Rose
- WC Rose
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- WH Adolph
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- WW Campbell
- Y Armand
- Y Kotake
- Y Saho
- Publication venue
- 'Springer Science and Business Media LLC'
- Publication date
- 01/01/1950
- Field of study
Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives
- Author
- A Aiuti
- A Aiuti
- A Aiuti
- A Aiuti
- A Biffi
- A Consiglio
- A Gogol-Doring
- A Hua-Van
- A Ramezani
- A Stasi Di
- AG Uren
- AM Geurts
- Amilcar Tanuri
- Andrés Delgado-Cañedo
- AR Schroder
- AT Das
- B Moldt
- BA Brody
- BD Brown
- BL Levine
- BS Sundarasetty
- C Bonini
- C Cattoglio
- CC Bouuaert
- CE Dunbar
- CM Kowolik
- CN Barese
- D Weissman
- D Zychlinski
- DA Williams
- DB Kohn
- DB Kohn
- DB Kohn
- DL Galvan
- DL Porter
- E Kowarz
- E Montini
- EL Aronovich
- EL Kuether
- ER Burnight
- F Bushman
- F Candotti
- FD Bushman
- G Liu
- G Luo
- G Turchiano
- H Goulaouic
- H Miyatake
- H Nakanishi
- H Singh
- H Singh
- H Yin
- H Zayed
- HB Gaspar
- HB Gaspar
- I Grabundzija
- I Grabundzija
- J Baus
- J Cadinanos
- J Kaiser
- J Karn
- J Matrai
- JA Bueren
- JB Owens
- JM Wilson
- José Eduardo Vargas
- JR Ohlfest
- JS Park
- JW Rausch
- K Kaji
- K Katter
- K Ladell
- K Pauwels
- K Voigt
- K Woltjen
- K Yusa
- K Yusa
- KC Straathof
- KT Roybal
- L Chicaybam
- L Chicaybam
- L Chicaybam
- L Mates
- L Naldini
- L Naldini
- LC Cary
- Leonardo Chicaybam
- M Bellodi-Privato
- M Cavazzana
- M Cavazzana-Calvo
- M Cavazzana-Calvo
- M Galla
- M Gaszner
- M Kalos
- M Yamashita
- MA Kotterman
- MA Li
- MA Li
- Martin H. Bonamino
- MD Li
- ME Morales
- MH Malim
- MH Wilson
- MJ Fraser
- ML Suster
- MO Sauvain
- MP Cicalese
- MT Lupo-Stanghellini
- MV Evans-Galea
- N Cartier
- N Hanoun
- N Kacherovsky
- N Sharma
- O Delelis
- O Negre
- O Walisko
- OW Merten
- P Kebriaei
- P Kebriaei
- P Lin
- P Maier
- PB Hackett
- PD Peng
- PI Arumugam
- PV Manuri
- R Greco
- RA Katz
- Renato Tetelbom Stein
- RM Blaese
- RM Welsh
- RR MacGregor
- RV Guntaka
- S Bire
- S Ding
- S Galanie
- S Hacein-Bey-Abina
- S Hacein-Bey-Abina
- S Hacein-Bey-Abina
- S Joshi
- S Nayak
- S Newrzela
- SJ Howe
- SK Maurya
- SR Kumar
- SR Yant
- SR Yant
- SR Yant
- T VandenDriessche
- W Garrels
- W Garrels
- W Wang
- WS Lynn
- X Huang
- X Wu
- X Xue
- YE Yu
- YW Zhang
- Z Ivics
- Z Ivics
- Z Ivics
- Z Izsvak
- Publication venue
- 'Springer Science and Business Media LLC'
- Publication date
- Field of study
Die Pathologie der Avitaminosen und Hypervitaminosen
- Author
- A Albeggiani
- A Aschkenasy
- A Bennandi
- A Brown
- A Brusa
- A Carlsson
- A Carotene
- A Chevallier
- A Chevallier
- A Corcos
- A Cruchaud
- A Dagianti
- A Dury
- A Faller
- A Faller
- A Ferraro
- A Fingerland
- A Forse
- A Froehlich
- A Gellhorn
- A Gellhorn
- A Gerber
- A Gerber
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Giroud
- A Golden
- A Grimm
- A Heise
- A Hirt
- A Hoffer
- A Holst
- A Holst
- A Ingelmansundberg
- A Ingelmansundberg
- A Issidorides
- A JuhaszschÄFfer
- A JuhaszschÄFfer
- A Kirschbaum
- A Kornberg
- A Kornberg
- A Kornberg
- A Kornberg
- A Krupski
- A Krupski
- A Libretti
- A Mariakulandai
- A Matet
- A Mex
- A Minkowski
- A Minkowski
- A Morelli
- A MUsIni
- A Narrozzi
- A Narrozzi
- A Pirie
- A Pirie
- A Pittoni
- A Prader
- A Schleich
- A Sesmarais
- A Steiger
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Studer
- A Szentgyorgy
- A Tannenbaum
- A Tannenbaum
- A Taylor
- A Taylor
- A Terbruggen
- A Volta
- A Werthemann
- A Werthemann
- A Windaus
- A Wolf
- A Wolf
- A. H. Douthwaite
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AA Nelson
- AB Benchimol
- AB Kar
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- AB Vogelsang
- AB Watts
- AB Watts
- AC Allison
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- AC Silva
- AC Upton
- AC Upton
- AC Upton
- AC Wiese
- AC Wiese
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- AD Gillanders
- AD Laurence
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- AE Axelrod
- AE Axelrod
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- AN Drury
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- AO Seeler
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- AS Gordon
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- B Green
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- C Artom
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- SE Walker
- SG Ross
- SG Smith
- SG Smith
- SJ Hartfall
- SJ Piliero
- SK Elster
- SQ Cohlan
- SQ Cohlan
- SQ Cohlan
- SQ Conman
- SR Ames
- SR Ames
- SR Ames
- SR Ames
- SR Ames
- SS Chernick
- SS Chernick
- SS Spicer
- SS Spicer
- SS Turesky
- SS Turesky
- SS Turesky
- SW Clausen
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SW Lippincott
- SY Pan
- T Barlow
- T Barlow
- T Barlow
- T Gillman
- T Gordonoff
- T Gordonoff
- T Iwabuchi
- T Masella
- T Moore
- T Moore
- T Moore
- T Moore
- T Terroine
- TA Jukes
- TC Jones
- TC Lonie
- TC Wyatt
- TD Luckey
- TD Spies
- TD Spies
- TE Metre
- TF Zucker
- TH Brandt
- TH Jukes
- TH Tomlinson
- TH Tomrinson
- TJ Cunha
- TJ Cunha
- TJ Haley
- TK Oliver
- TL Miller
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TM Ferguson
- TR Riggs
- TS Argyris
- TW Gullickson
- TW Gullicxson
- Untersuchungen über die Wirksamkeit des Vitamins BB (Pyridoxin) bei diphtherischen Lähmungen
- V Claassen
- V Demole
- V Demole
- V Demole
- V Demole
- V Demole
- V Piccioni
- V Ramalingasfami
- V Ramalingaswami
- V Ramalingaswami
- V Ramalingaswami
- V Ramalingaswami
- V Spampinato
- V Spampinato
- V. L. Hart
- VA Drill
- VD Vigneaud
- VE Kinsey
- VN Patfardhan
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Antopol
- W Bartley
- W Bartley
- W Becker
- W Birkmayer
- W Birkmayer
- W Bolagna
- W Boltz
- W Burkl
- W Burroughs
- W Burroughs
- W Creutzfeld
- W Daubenmerkl
- W Daubenmerkl
- W Dittrich
- W Eger
- W Ehrengut
- W Ehrengut
- W Fey
- W Goettscii
- W Hessler
- W Hohlweg
- W Hohlweg
- W Kircher
- W Kwiatkowski
- W Landauer
- W Landauer
- W Loffler
- W Marusich
- W Mcfarland
- W Montagna
- W Montagna
- W Muller
- W Neuweiler
- W Neuweiler
- W Neuweiler
- W Neuweiler
- W Neuweiler
- W Neuweiler
- W Neuweiler
- W Neuweiler
- W Rozynek
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Ruppel
- W Salmon
- W Sebrell
- W Stepp
- W Stepp
- W Stepp
- W Swoboda
- W Tobler
- W Trager
- W Trager
- W. E. Shute
- W. F. Alexander
- W. W. Fox
- WA Grimes
- WA Krehl
- WA Krehl
- WB Atkinson
- WB Atkinson
- WB Bean
- WB Hawkins
- WB Leftwich
- WB Mcallister
- WB Robertson
- WB Robertson
- WB Robertson
- WB Robertson
- WB Robertson
- WC Aalsmeer
- WC Owens
- WC Russell
- WC Thomas
- WD King
- WD Salmon
- WD Salmon
- WD Salmon
- WD Salmon
- WE Germeraad
- WE Germeraad
- WE Loch
- WE Phillips
- WE Shute
- WF Abercrombie
- WF Orr
- WF Perry
- WG Casselman
- WG Casselman
- WG Hoekstra
- WG Hoekstra
- WG Jaffe
- WG Jaffe
- WG Jaffe
- WG Jaffe
- WH Eddy
- WH Fishman
- WH Fishman
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Griffith
- WH Jr
- WH Sebrell
- WH Sebrell
- WH Sebrell
- WH Sebrell
- WJ Darby
- WJ Emerson
- WJ Griffiths
- WJ Mcganity
- WK Hall
- WK Manning
- WL Money
- WL Money
- WL Money
- WL Williams
- WM Booker
- WM Booker
- WO Brown
- WO Motte
- WO Russell
- WO Russell
- WO Russell
- WP Covell
- WP Covell
- WP Lehrer
- WR Ruegamer
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WS Hartroft
- WT Wong
- WV Drigalski
- WW Cravens
- WW Cravens
- WW Cravens
- WW Cravens
- WW Crnvens
- WW Crnvens
- WW Crnvens
- WW Hawkins
- WW Hawkins
- WW Hawkins
- WW Hawkins
- WW Wainwright
- Y Hosoya
- Y Raoul
- YO Chang
- Z Menschik
- Z Menschik
- Z Menschik
- Z Menschir
- ZM Bacq
- Publication venue
- 'Springer Science and Business Media LLC'
- Publication date
- 01/01/1962
- Field of study
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
- Author
- A Angelis
- A Annoni
- A Biffi
- A Cantore
- A Dhawan
- A Doenecke
- A Donsante
- A Ehrhardt
- A Ehrhardt
- A Fischer
- A Kiang
- A Mian
- A Mullard
- A Nowrouzi
- A Rosewell Shaw
- AA Rahim
- AC Nathwani
- AC Nathwani
- AC Nathwani
- AH Chang
- AK Zaiss
- AM Maguire
- AM Puumalainen
- AW Nienhuis
- B Crane
- B Thaci
- BD Brown
- BE Deverman
- C Booth
- C Bordignon
- C Kaeppel
- C Li
- C Mah
- C Summerford
- C Unzu
- CN Mattar
- CS Manno
- CS Manno
- D Duan
- D D’Avola
- D Majhen
- DA Muruve
- DD Koeberl
- DG Miller
- DM McCarty
- DM McCarty
- DP Rastall
- E Basner-Tschakarjan
- E Breous
- E Dolgin
- E Hastie
- EL Kuether
- EM Sokal
- EM Wilson
- EP Armstrong
- F Liu
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Mingozzi
- F Schmitt
- F Vigant
- FD Ledley
- G Buchlis
- G Schiedner
- GC Pien
- GE Berry
- GJ McGarrity
- GP Gao
- GS Lipshutz
- H Azuma
- H Buning
- H Jiang
- H Jiang
- H Miyoshi
- HG Zhang
- HM Viecelli
- I Gil-Farina
- I Trapani
- Ian E. Alexander
- IM Verma
- J Haberle
- J Hoggatt
- J Lu
- J Pasi
- J Tang
- J Zhu
- JA Wolff
- JC Grieger
- JC Nault
- JE Rabinowitz
- JH McIntosh
- JL Andrews
- JM Coppoletta
- JM Wilson
- JN Lozier
- JR Counsell
- JS Powell
- Julien Baruteau
- JW Bainbridge
- K Benihoud
- K Jooss
- K Vercauteren
- KI Berns
- KM Flanigan
- L Apolonia
- L Cassiday
- L Couto
- L Lisowski
- L Sendra
- L Wang
- LF Earley
- LG Chicoine
- LM Belalcazar
- LM Kattenhorn
- LV Tse
- M Cavazzana-Calvo
- M Cerreto
- M Cooper
- M Elsabahy
- M Grossman
- M Grossman
- M Li
- M Naumer
- M Spada
- M Suzuki
- MA Kay
- MA Kay
- MA Kay
- MA Kotterman
- MK Chuah
- ML Brantly
- ML Hirsch
- N Bessis
- N Brunetti-Pierri
- N Brunetti-Pierri
- N Brunetti-Pierri
- N Brunetti-Pierri
- N Cartier
- N Junge
- N Morral
- N Touchot
- P Bell
- P Mayrhofer
- P Monahan
- P Piccolo
- Paul Gissen
- PE Monahan
- PS Reddy
- PT Clayton
- R Alba
- R Calcedo
- R Castello
- R Condiotti
- R Gebhardt
- R Gramignoli
- R Macaulay
- R Sarkar
- R Thwaite
- R Zufferey
- RD Hickey
- RJ Chandler
- RJ Chandler
- RJ Chandler
- RJ Yanez-Munoz
- RM Blaese
- RW Atchison
- RW Herzog
- S Boutin
- S Chira
- S Hacein-Bey-Abina
- S Hacein-Bey-Abina
- S Mukherjee
- S Nomura
- S Pillay
- SA Fahs
- SC Cunningham
- SC Cunningham
- SC Cunningham
- SE Raper
- SE Raper
- SE Raper
- SE Raper
- SH Orkin
- Simon N. Waddington
- SJ Nightingale
- SL Ginn
- SL Stephen
- SN Waddington
- SR Choudhury
- T Koo
- T Suwanmanee
- T Suzuki
- T Wirth
- T Yokoo
- TH Nguyen
- TR Flotte
- TR Flotte
- TR McKay
- V Louis Jeune
- V Monteilhet
- VR Arruda
- W Miesbach
- WM McCormack Jr
- WS Wold
- X Hou
- Y Chen
- Y Oishi
- Y Shi
- Y Yang
- Y Yang
- YS Tseng
- Publication venue
- 'Springer Science and Business Media LLC'
- Publication date
- Field of study