유전자 치료

3. 세포의 종류

유전자 치료는 적용되는 세포의 종류에 따라 체세포 유전자 치료, 생식세포 유전자 치료, 줄기세포 유전자 치료로 분류할 수 있다.

• 체세포 유전자 치료 (SCGT): 배우자, 생식 세포, 생식 모세포, 미분화 줄기 세포를 제외한 체세포에 치료 유전자를 도입한다. 유전체 변형은 개인에게만 영향을 미치고 자손에게는 유전되지 않는다.
• 생식세포 유전자 치료 (GGT): 정자나 난자와 같은 생식 세포에 기능 유전자를 넣어 유전체를 변형시킨다. 변형된 유전자는 해당 생물체의 모든 세포에 포함되며, 유전 가능하여 다음 세대로 전달된다.
• 줄기세포 유전자 치료: 자기 재생 능력이 있는 줄기세포를 이용하므로 치료 효과가 오래 지속되어 주기적인 치료가 필요 없다는 장점이 있다.

3. 1. 체세포 유전자 치료 (SCGT)

3. 2. 생식세포 유전자 치료 (GGT)

3. 3. 줄기세포 유전자 치료

4. 유전자 전달체/운반체

유전자 전달체는 치료 유전자를 세포 내로 운반하는 역할을 한다. 유전자 전달은 여러 방법으로 달성할 수 있다. 크게 두 가지로 분류하면 재조합 바이러스(바이러스 벡터)와 나체 DNA 또는 DNA 복합체(비바이러스 방법)이다.^[63]

초기에는 원하는 단백질을 발현시키는 유전자를 투여하는 데에 중점을 두었으나, 기술이 발전하면서 3세대 유전자 가위인 CRISPR 등을 사용하여 직접 유전체를 편집하는 방향으로 연구가 진행되었다. 바이러스 벡터는 환자의 유전체에 유전자를 직접 도입하여, 원하는 단백질을 발현시키거나 핵산 분해효소(CRISPR 등)를 발현시켜 치료 효과를 얻도록 한다. 1980년대 후반부터 관련 연구가 꾸준히 진행되었고, 유전자 가위를 이용한 유전자 치료는 2010년대에 보고되기 시작했다.^[326]

4. 1. 바이러스 벡터

4. 2. 비 바이러스 벡터

5. ''Ex vivo''와 ''In vivo''의 차이점

일반적으로 유전자 치료제는 체외(''ex vivo'')와 체내(''in vivo'')로 구분된다.^[53][54] 생체 외(ex vivo) 유전자 치료는 환자의 세포를 채취하여 유전자를 변형시킨 후 다시 환자에게 주입하는 방식이고, 생체 내(in vivo) 유전자 치료는 유전자를 운반하는 벡터를 환자에게 직접 주입하는 방식이다. 1990년 아샨티 데실바가 아데노신 탈아미나제 결핍증(ADA-SCID) 치료를 받으면서 유전자 치료가 성공적으로 받아들여졌다.

5. 1. 생체 내 ''In vivo'' 유전자 치료제

5. 2. 생체 외 ''Ex vivo'' 유전자 치료제

• '''장점:'''
• * '오프 타깃 효과' 감소: ''In vivo'' 유전자 치료의 단점인 '오프 타깃 효과'를 줄일 수 있다.
• * 면역 반응 감소: 바이러스 전달체에 의해 유발될 수 있는 면역 반응을 최소화할 수 있다.
• * 세포 선별, 증식, 품질 평가 가능: 형질 전환된 세포에 대해 선별, 증식, 품질 평가를 수행하여 안전성과 효능을 높일 수 있다.
• '''활용:'''
• * CAR-T 치료법: CAR-T 치료법과 같은 ''생체 외 유전자 치료''에서는 환자 자신의 세포(자가 세포) 또는 건강한 공여 세포(동종 세포)를 특정 단백질(예: 키메라 항원 수용체)을 발현하도록 벡터를 사용하여 신체 외부에서 수정한다.

6. 한계

유전자 치료는 아직 해결해야 할 문제들이 많다.

1990년대에 임상 시험이 시작된 이후, 유전자 치료 연구는 여러 문제로 인해 쇠퇴와 부흥을 반복했다. 1999년에는 사망 사고가 발생했으며, 백혈병을 앓게 된 환자도 있었다. 이러한 이유로, ES 세포나 iPS 세포를 중심으로 한 줄기 세포 치료로 연구 방향이 전환되기도 했다.^[318]

2010년대에 들어 징크 핑거 뉴클레아제 (ZFN), TALEN, CRISPR/Cas9를 중심으로 한 게놈 편집 기술이 등장했다.^[305] 2015년에는 중화인민공화국에서 CRISPR/Cas9를 사용해 세계 최초로 인간 수정란 유전자 조작을 시도하여 국제적인 논란이 일었다.^[306][307] 이는 인간 수정란에 대한 게놈 편집 기술의 생명 윤리적 규제에 대한 새로운 과제를 제기했다.^[308]

유전자 치료의 일반적인 문제점은 다음과 같다.

• 전달 문제: DNA는 전하를 띤 분자이므로 세포막과 핵막을 통과하기 어렵다. 바이러스를 벡터로 사용하기도 하지만, 제어가 쉽지 않다.
• 기능 문제: DNA를 표적 세포에 넣더라도, 단백질로 전사 및 번역될 가능성이 낮아 기능 발현이 어려울 수 있다.
• 면역 반응 문제: 바이러스는 이물질로 간주되어 면역계의 공격을 받을 수 있으므로, 장기간의 바이러스 투여가 어렵다.

6. 1. 짧은 치료 효과 기간

6. 2. 면역 반응

6. 3. 다발 장애

6. 4. 삽입 돌연변이 유발

6. 5. 비용

6. 6. 윤리적 문제

2015년에는 중화인민공화국에서 CRISPR/Cas9 기술을 이용해 인간 수정란 유전자 조작을 시행하여 국제적인 논란이 일기도 했다.^[306][307] 이는 인간 수정란에 대한 게놈 편집 기술의 생명 윤리적 규제 문제를 제기한다.^[308]

7. 사용 전망

유전자 치료는 질병 치료뿐만 아니라 운동 능력, 외모, 신진대사, 기억력, 지능 등 다양한 인간의 능력을 향상시키는 데 사용될 수 있다는 전망이 제기되고 있다.

유전자 도핑은 아직까지 발생 사례는 없지만, 운동선수들이 유전자 치료 기술을 사용할 가능성에 대한 우려가 존재한다.^[130] 일각에서는 모든 선수에게 유전자 도핑이 동등하게 제공된다면 공정한 경쟁이 가능하다는 주장도 있지만,^[131] 윤리적인 문제를 제기하는 비판적인 시각도 존재한다.

인간 유전공학, 특히 생식세포 유전자 조작에 대한 윤리적 논쟁은 1990년대부터 꾸준히 제기되어 왔다.^[456] 태아가 유전자 변형되지 않은 채로 태어날 권리, 부모가 자녀의 유전자를 선택할 권리 등 다양한 주장이 대립하고 있다.^{[452][453][454]} CRISPR와 같은 최신 기술의 등장은 이러한 논쟁을 더욱 심화시키고 있으며, 2015년에는 과학자들이 인간 생식세포 유전체 변형에 대한 일시적 중단(모라토리움)을 제안하기도 했다.^[424][425]

미국 국립 과학 아카데미와 국립 의학 아카데미 위원회는 유전체 편집의 안정성과 효능이 보장되고, 대상이 심각한 상황에 처해 있으며, 엄격한 통제가 가능한 경우에만 인간 유전체 편집을 지지한다는 입장을 밝혔다.^[461] 미국 의학 협회의 윤리 및 사법위원회는 특정 형질을 강화하는 유전자 조작은 매우 제한적인 상황에서만 허용되어야 하며, 다음과 같은 조건을 충족해야 한다고 주장했다.^[455]

7. 1. 유전자 도핑

7. 2. 인간 유전공학

8. 고려할 점

이론적으로 인간과 다른 동물에게 유전자를 주입하는 것에는 큰 차이가 없다. 그러나 여기에는 복잡한 윤리적, 기술적 문제점이 따른다.^[174][219]

열성 유전병과 우성 유전병을 치료하는 방법에는 기본적인 기술적 차이점이 있다. 열성 유전병은 특정 생성물의 결핍에 의해 나타나기 때문에 온전한 생성물 유전자를 생물체에 주입하면 된다. 이와 달리 우성 유전병의 경우에는 결함 유전자를 정상 상태로 전환시키거나 비활성화시켜야 한다.

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_[93] 간행물 FDA Approves First Gene Therapy for Treatment of Aromatic L-amino Acid Decarboxylase Deficiency https://www.fda.gov/[...] 2024-11-14
_[94] 웹사이트 Upstaza EPAR https://www.ema.euro[...] 2022-04-13
_[95] 웹사이트 Casgevy https://www.fda.gov/[...] 2023-12-08
_[96] 웹사이트 Abecma (idecabtagene vicleucel) https://www.fda.gov/[...] U.S. Food and Drug Administration (FDA) 2021-04-21
_[97] 웹사이트 Breyanzi (lisocabtagene maraleucel) https://www.fda.gov/[...] U.S. Food and Drug Administration (FDA) 2021-03-04
_[98] 웹사이트 Lyfgenia https://www.fda.gov/[...] 2023-12-08
_[99] 간행물 FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer https://www.fda.gov/[...] 2022-12-16
_[100] 웹사이트 FDA approves obecabtagene autoleucel for acute lymphoblastic leukemia https://www.fda.gov/[...] 2024-11-08
_[101] 간행물 Autolus Therapeutics Announces FDA Approval of Aucatzyl (obecabtagene autoleucel – obe-cel) for adults with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) https://www.globenew[...] Autolus Therapeutics 2024-11-08
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_[104] 웹사이트 FDA Approves T-VEC to Treat Metastatic Melanoma https://www.cancer.g[...] 2015-11-25
_[105] 웹사이트 Imlygic https://www.ema.euro[...] 2018-09-17
_[106] 간행물 First gene therapy to treat severe haemophilia A https://www.ema.euro[...] EMA 2022-06-26
_[107] 웹사이트 Roctavian: Pending EC decision https://www.ema.euro[...] EMA 2022-06-26
_[108] 웹사이트 Roctavian https://ec.europa.eu[...] 2022-09-06
_[109] 간행물 FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss https://www.fda.gov/[...] FDA 2021-07-23
_[110] 웹사이트 Luxturna https://www.ema.euro[...] EMA 2018-09-24
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_[144] 뉴스 Chinese Scientists Edit Genes of Human Embryos, Raising Concerns https://www.nytimes.[...] 2015-04-24
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_[159] 뉴스 First patient treated in stem cell study https://www.washingt[...] 2010-11-10
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_[161] 뉴스 Gene Therapy Ordered Halted At University https://www.nytimes.[...] 2010-11-10
_[162] 웹사이트 Murine Gene Therapy Corrects Symptoms of Sickle Cell Disease http://www.the-scien[...] 2010-08-17
_[163] 웹사이트 Gene Therapy Corrects Sickle Cell Disease In Laboratory Study https://www.scienced[...] 2008-12-04
_[164] 웹사이트 Subtle gene therapy tackles blood disorder https://www.newscien[...] 2010-08-17
_[165] 웹사이트 DNA nanoballs boost gene therapy https://www.newscien[...] 2010-08-17
_[166] 웹사이트 Undercover genes slip into the brain https://www.newscien[...] 2010-08-17
_[167] 웹사이트 Gene therapy may switch off Huntington's https://www.newscien[...] 2010-08-17
_[168] 논문 Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 http://www.medscape.[...] 2006-04
_[169] 논문 Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer 2006-05
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_[172] 웹사이트 Penn Medicine presents HIV gene therapy trial data at CROI 2009 http://www.eurekaler[...] 2009-11-19
_[173] 뉴스 Gene therapy first for poor sight http://news.bbc.co.u[...] 2010-05-03
_[174] 논문 Safety and efficacy of gene transfer for Leber's congenital amaurosis 2008-05
_[175] 논문 Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration 2010-03
_[176] 논문 Vision 1 year after gene therapy for Leber's congenital amaurosis 2009-08
_[177] 논문 Effect of gene therapy on visual function in Leber's congenital amaurosis 2008-05
_[178] 논문 Colour blindness corrected by gene therapy
_[179] 논문 Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy https://hal-cea.arch[...] 2009-11
_[180] 논문 Gene therapy rescues cone function in congenital achromatopsia 2010-07
_[181] 논문 Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia 2010-09
_[182] 논문 Beta-thalassemia 2010-05
_[183] 웹사이트 Gene Therapy Frees Beta-Thalassemia Patient From Transfusions for 2+ Years http://www.medscape.[...] 2010-09-16
_[184] 논문 Five year outcome of lentiviral gene therapy for human beta-thalassemia, lessons and prospects http://www.pagepress[...] 2013-03-20
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_[188] 간행물 The Man Who Was Cured of HIV and What It Means for a Cure for AIDS https://nymag.com/he[...] 2011-05-27
_[189] 웹사이트 Letters from the Director: Remembering Timothy Ray Brown: A Champion for HIV Cure Research https://www.oar.nih.[...] 2022-06-26
_[190] 논문 Cell therapy fights leukaemia
_[191] 웹사이트 Gene Therapy Turns Several Leukemia Patients Cancer Free. Will It Work for Other Cancers, Too? http://singularityhu[...] Singularity Hub 2014-01-06
_[192] 논문 Phase I clinical trial on intracoronary administration of Ad-hHGF treating severe coronary artery disease 2009-07
_[193] 논문 Enhanced cardioprotective effects by coexpression of two isoforms of hepatocyte growth factor from naked plasmid DNA in a rat ischemic heart disease model 2011-10
_[194] 웹사이트 Neovasculogen http://www.eurolab.u[...]
_[195] 논문 pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia 2015-09
_[196] 웹사이트 On Cancer: Launch of Stem Cell Therapy Trial Offers Hope for Patients with Inherited Blood Disorder http://www.mskcc.org[...] Memorial Sloan-Kettering Cancer Center 2012-07-16
_[197] 웹사이트 European Agency Recommends Approval of a Gene Therapy https://www.nytimes.[...] 2012-07-20
_[198] 웹사이트 Gene Therapy Arrives in Europe http://www.the-scien[...] 2012-11-06
_[199] 웹사이트 Gene therapy: Glybera approved by European Commission https://www.bbc.co.u[...] 2012-11-02
_[200] 간행물 First Gene Therapy Approved by European Commission https://www.uniqure.[...] 2012-11-02
_[201] 웹사이트 Chiesi and uniQure delay Glybera launch to add data http://www.thepharma[...] The Pharma Letter 2014-08-04
_[202] 뉴스 First gene therapy drug sets million-euro price record https://www.reuters.[...] 2014-11-26
_[203] 뉴스 Gene-Therapy Approval Marks Major Milestone https://www.wsj.com/[...] 2012-11-02
_[204] 논문 $1-million price tag set for Glybera gene therapy 2015-03-03
_[205] 뉴스 Gene therapy approved in Europe for first time http://www.bionews.o[...]
_[206] 뉴스 The World's Most Expensive Medicine Is a Bust https://www.technolo[...] 2016-05-04
_[207] 웹사이트 Gene therapy cures leukaemia in eight days https://www.newscien[...] 2013-03-26
_[208] 웹사이트 Pioneering gene therapy trials offer hope for heart patients https://www.theguard[...] 2013-04-30
_[209] 웹사이트 Cardiac gene therapy news: The Physicians Clinic https://www.thephysi[...] 2013-09-08
_[210] 웹사이트 Celladon Receives Breakthrough Therapy Designation From FDA for Mydicar, Novel, First-in-Class Therapy in Development to Treat Heart Failure http://ir.celladon.c[...] 2015-07-10
_[211] 논문 Gene therapy: No improvement in outcomes with gene therapy for heart failure 2016-03
_[212] 논문 Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy https://www.openacce[...] 2013-08
_[213] 논문 Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott–Aldrich syndrome 2013-08
_[214] 웹사이트 Gene therapy: 'Tame HIV' used to cure disease https://www.bbc.co.u[...] 2015-04-21
_[215] 논문 An emerging era of clinical benefit from gene therapy 2015-04
_[216] 웹사이트 Gene therapy cure for children with 'bubble baby' disease https://www.scienced[...] 2014-11-18
_[217] 웹사이트 'Bubble kid' success puts gene therapy back on track https://www.newscien[...] 2013-10-30
_[218] 웹사이트 Gene therapy provides safe, long-term relief for patients with severe hemophilia B https://www.scienced[...] 2014-11-20
_[219] 논문 Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial 2014-03
_[220] 웹사이트 Gene therapy restores sight in people with eye disease https://www.newscien[...] 2014-01-25
_[221] 논문 Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV 2014-03
_[222] 웹사이트 Scientists Create Genetically Modified Cells That Protect Against HIV http://io9.com/scien[...] 2014-03-06
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_[226] 뉴스 Protection Without a Vaccine https://www.nytimes.[...] 2015-03-09
_[227] 논문 AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges 2015-03
_[228] 뉴스 Scientists Seek Ban on Method of Editing the Human Genome https://www.nytimes.[...] 2015-03-19
_[229] 뉴스 A Powerful New Way to Edit DNA https://www.nytimes.[...] 2015-03-03
_[230] 논문 Biotechnology. A prudent path forward for genomic engineering and germline gene modification 2015-04
_[231] 논문 Don't edit the human germ line 2015-03
_[232] 뉴스 Scientists Place Moratorium on Edits to Human Genome That Could Be Inherited https://www.nytimes.[...] 2015-12-03
_[233] 뉴스 Gene editing: Is era of designer humans getting closer? https://www.bbc.co.u[...] BBC News Health 2015-12-03
_[234] 웹사이트 Genetically Altered Skin Saves A Boy Dying Of A Rare Disease https://www.npr.org/[...]
_[235] 뉴스 Leukaemia cure hopes rise as girl is gene‑edited http://www.thetimes.[...] 2017-02-19
_[236] 뉴스 Baby girl is first in the world to be treated with 'designer immune cells' https://www.theguard[...] 2015-11-05
_[237] 뉴스 'Designer cells' reverse one-year-old's cancer https://www.bbc.com/[...] 2015-11-05
_[238] 웹사이트 Summary of opinion1 (initial authorisation) Strimvelis http://www.ema.europ[...] 2016-04-01
_[239] 뉴스 Europe gives green light to first gene therapy for children http://www.reuters.c[...] 2016-04-01
_[240] 웹사이트 Second gene therapy wins approval in Europe http://www.bionews.o[...] 2016-06-06
_[241] 간행물 Gene Therapy Approved https://www.newscien[...] 2016-04-09
_[242] 논문 Chinese scientists to pioneer first human CRISPR trial 2016-07
_[243] 뉴스 Chinese Scientists Become First to Use CRISPR Gene-Editing on Humans http://www.popularme[...] 2016-11-15
_[244] 논문 Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease 2016-06
_[245] 뉴스 New gene therapy 'shrinks tumours like ice cubes' http://www.thetimes.[...] 2017-03-01
_[246] 간행물 Gene therapy 'cures' boy of blood disease that affects millions https://www.newscien[...] 2017-03
_[247] 간행물 FDA approval brings first gene therapy to the United States https://www.fda.gov/[...] U.S. [[Food and Drug Administration]] (FDA) 2017-08-30
_[248] 웹사이트 FDA approves axicabtagene ciloleucel for large B-cell lymphoma https://www.fda.gov/[...] 2018-01-05
_[249] Youtube DIY Human CRISPR Myostatin Knock-Out https://www.youtube.[...] 2020-05-20
_[250] 웹사이트 The First Attempt At Human CRISPR Gene Editing http://www.josiahzay[...] 2020-05-20
_[251] 뉴스 AP Exclusive: US scientists try 1st gene editing in the body https://apnews.com/a[...] 2017-11-15
_[252] 논문 A human has been injected with gene-editing tools to cure his disabling disease. Here's what you need to know 2017-11
_[253] 웹사이트 The First Man to Have His Genes Edited Inside His Body https://www.theatlan[...] 2020-11-17
_[254] 논문 First test of in-body gene editing shows promise 2018-09-05
_[255] 뉴스 Tests suggest scientists achieved 1st 'in body' gene editing https://www.apnews.c[...] 2019-02-07
_[256] 웹사이트 Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II https://clinicaltria[...] "[[U.S. National Library of Medicine]]" 2019-02-07
_[257] 논문 AAV5-Factor VIII Gene Transfer in Severe Hemophilia A 2017-12
_[258] 논문 A Cure for Hemophilia within Reach 2017-12
_[259] 간행물 FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss https://www.fda.gov/[...] 2017-12-20
_[260] 뉴스 Spark Therapeutics Sets Price Of Blindness-Treating Gene Therapy At $850,000 https://www.forbes.c[...] 2018-01-04
_[261] 뉴스 The FDA approved a gene therapy to treat blindness in a groundbreaking moment for DNA-based medicine http://www.newsweek.[...] 2017-12-20
_[262] 뉴스 At $2.1 Million, New Gene Therapy Is The Most Expensive Drug Ever https://www.npr.org/[...] 2019-05-24
_[263] 뉴스 EU stamps historic OK on bluebird's gene therapy for β-thalassemia – now sit back and wait for the price https://endpts.com/e[...] 2019-06-03
_[264] 웹사이트 Zynteglo EPAR https://www.ema.euro[...] 2020-06-08
_[265] 웹사이트 Single Ascending Dose Study in Participants With LCA10 https://clinicaltria[...] 2019-08-20
_[266] 웹사이트 Allergan and Editas Medicine Initiate the Brilliance Phase 1/2 Clinical Trial of AGN-151587 (EDIT-101) for the Treatment of LCA10 http://ir.editasmedi[...] 2019-08-20
_[267] 논문 CRISPR treatment inserted directly into the body for first time 2020-03
_[268] 뉴스 In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder https://www.npr.org/[...] 2019-07-29
_[269] 웹사이트 Zolgensma EPAR https://www.ema.euro[...] 2020-10-16
_[270] 웹사이트 Audentes Therapeutics Provides Update on the ASPIRO Clinical Trial Evaluating AT132 in Patients with X-linked Myotubular Myopathy https://www.audentes[...] 2020-09-21
_[271] 뉴스 Astellas' Audentes reports 3rd death in gene therapy trial https://www.fiercebi[...] 2020-08-21
_[272] 논문 High-dose AAV gene therapy deaths 2020-08-01
_[273] 웹사이트 Libmeldy: Pending EC decision https://www.ema.euro[...] 2020-10-16
_[274] 웹사이트 New gene therapy to treat rare genetic disorder metachromatic leukodystrophy https://www.ema.euro[...] 2020-10-16
_[275] 웹사이트 Libmeldy EPAR https://www.ema.euro[...] 2020-12-22
_[276] 웹사이트 Lysogene provides update on the AAVance Clinical Trial Evaluating LYS-SAF302 in Patients with MPS IIIA – Lysogene https://www.lysogene[...] 2020-10-17
_[277] 논문 Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency 2021-05
_[278] 웹사이트 AIDS virus used in gene therapy to fix 'bubble baby' disease https://www.statnews[...] 2021-07-19
_[279] 웹사이트 Gene therapy restores immune function in children with rare immunodeficiency https://www.nih.gov/[...] 2021-07-19
_[280] 웹사이트 Why Gene Therapy Caused Leukemia In Some 'Boy In The Bubble Syndrome' Patients https://www.scienced[...] 2021-07-19
_[281] 논문 Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1 2019-04
_[282] 뉴스 HIV used to cure 'bubble boy' disease https://www.bbc.com/[...] 2021-07-19
_[283] 웹사이트 These Scientists May Have Found a Cure for 'Bubble Boy' Disease https://www.smithson[...] 2021-07-19
_[284] 웹사이트 Gene therapy restores immunity in infants with rare immunodeficiency disease https://www.nih.gov/[...] 2020-06-04
_[285] 논문 CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis 2021-08
_[286] 웹사이트 News: Clinical Trial Update: Positive Data for First Ever In Vivo CRISPR Medicine https://crisprmedici[...] 2021-12-16
_[287] 논문 The science news that shaped 2021: Nature's picks 2021-12
_[288] 논문 Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons 2021-07
_[289] 논문 Gene therapy restores missing dopamine in children with rare brain disease https://www.science.[...] 2021-07-18
_[290] 웹사이트 Gene therapy trial points to a wider window to alter course of rare disease https://www.statnews[...] 2021-07-18
_[291] 논문 AAV gene therapy for Tay-Sachs disease 2022-02
_[292] 웹사이트 First gene therapy for Tay-Sachs disease successfully given to two children http://theconversati[...] 2022-03-07
_[293] 웹사이트 Parents spark breakthrough gene therapy for children with Tay-Sachs disease https://www.independ[...] 2022-03-07
_[294] 웹사이트 Upstaza: Pending EC decision https://www.ema.euro[...] 2022-05-22
_[295] 간행물 PTC Therapeutics Receives Positive CHMP Opinion for Upstaza for the Treatment of AADC Deficiency https://ir.ptcbio.co[...] 2022-05-22
_[296] 논문 Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B https://discovery.uc[...] 2022-07
_[297] 웹사이트 Novel gene therapy could reduce bleeding risk for haemophilia patients https://www.scienced[...] 2022-08-03
_[298] 뉴스 Transformational therapy cures haemophilia B https://www.bbc.com/[...] 2022-08-03
_[299] 뉴스 Base editing: Revolutionary therapy clears girl's incurable cancer https://www.bbc.co.u[...] 2022-12-11
_[300] 웹사이트 Gene therapy eyedrops restored a boy's sight. Similar treatments could help millions https://apnews.com/a[...] 2023-07-31
_[301] 논문 An electrogenetic interface to program mammalian gene expression by direct current 2023-08-16
_[302] 웹사이트 PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy https://ir.ptcbio.co[...] 2024-11-21
_[303] 서적 遺伝子治療の現況と展望 http://www.jsog.or.j[...] 2015-12-15
_[304] 뉴스 ゲノム編集で難病治療／欧米勢、2～3年後にも／日本勢 モダリスが治験準備／安全証明・特許争い壁に https://www.nikkei.c[...] 日本経済新聞 2021-09-05
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_[314] 저널 Martin Cline loses appeal on NIH grant https://archive.org/[...] 1982-10
_[315] 서적 A Companion to Genethics https://books.google[...] John Wiley & Sons
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_[317] 뉴스 Gene therapy reverses sight loss and is long-lasting http://www.bbc.co.uk[...] BBC News, Science & Environment 2016-04-29
_[318] 논문 20 years of gene therapy for SCID 2010-06
_[319] 논문 Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency 2010-12
_[320] 논문 Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy 2010-07
_[321] 웹인용 Souped-up immune cells force leukaemia into remission https://www.newscien[...] 2013-04-15
_[322] 논문 AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial 2011-04
_[323] 논문 China approves first gene therapy 2004-01
_[324] 뉴스 Gene Therapy for PAD Approved http://www.dddmag.co[...] 2011-12-06
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_[334] 간행물 Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy 2017
_[335] 간행물 The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice 2016
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_[364] 웹인용 DNA nanoballs boost gene therapy https://www.newscien[...] 2002-05-12
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_[366] 웹인용 Gene therapy may switch off Huntington's https://www.newscien[...] 2003-03-13
_[367] 저널 Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1 http://www.medscape.[...] 2006-04
_[368] 저널 Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer 2006-05
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_[371] 웹인용 Penn Medicine presents HIV gene therapy trial data at CROI 2009 http://www.eurekaler[...] EurekAlert! 2009-02-10
_[372] 뉴스 Gene therapy first for poor sight http://news.bbc.co.u[...] BBC News 2007-05-01
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_[374] 저널 Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration 2010-03
_[375] 저널 Vision 1 year after gene therapy for Leber's congenital amaurosis 2009-08
_[376] 저널 Effect of gene therapy on visual function in Leber's congenital amaurosis 2008-05
_[377] 저널 Colour blindness corrected by gene therapy
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_[379] 저널 Gene therapy rescues cone function in congenital achromatopsia 2010-07
_[380] 저널 Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia 2010-09
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_[385] 저널 Cell therapy fights leukaemia
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_[387] 저널 Phase I clinical trial on intracoronary administration of Ad-hHGF treating severe coronary artery disease 2009-07
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_[389] 웹사이트 Vascular endothelial growth factor gene therapy - HSCI http://adisinsight.s[...] 2016-06-05
_[390] 웹사이트 Neovasculogen listing in Eurolab http://www.eurolab.u[...] 2015-08-04
_[391] 저널 pCMV-vegf165 Intramuscular Gene Transfer is an Effective Method of Treatment for Patients With Chronic Lower Limb Ischemia 2015-09
_[392] 웹인용 On Cancer: Launch of Stem Cell Therapy Trial Offers Hope for Patients with Inherited Blood Disorder http://www.mskcc.org[...] Memorial Sloan-Kettering Cancer Center 2012-07-16
_[393] 웹인용 Gene Therapy Arrives in Europe http://www.the-scien[...] 2012-11-06
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_[395] 웹사이트 First Gene Therapy Approved by European Commission http://www.uniqure.c[...] 2012-11-02
_[396] 웹인용 Chiesi and uniQure delay Glybera launch to add data http://www.thepharma[...] The Pharma Letter 2014-08-28
_[397] 뉴스 First gene therapy drug sets million-euro price record https://www.reuters.[...] 2014-11-26
_[398] 저널 Gene-Therapy Approval Marks Major Milestone https://www.wsj.com/[...] 2012-11-02
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_[400] 웹인용 Gene therapy approved in Europe for first time http://www.bionews.o[...] 2018-04-24
_[401] 뉴스 The World’s Most Expensive Medicine Is a Bust https://www.technolo[...] 2016-05-04
_[402] 웹인용 Gene therapy cures leukaemia in eight days https://www.newscien[...] 2013-03-26
_[403] 웹인용 Pioneering gene therapy trials offer hope for heart patients https://www.theguard[...] 2013-04-30
_[404] 웹사이트 First gene therapy trial for heart failure begins in UK https://www.thephysi[...] 2013-09-08
_[405] 웹사이트 Celladon Receives Breakthrough Therapy Designation From FDA for MYDICAR(R), Novel, First-in-Class Therapy in Development to Treat Heart Failure http://ir.celladon.c[...] 2014-04-10
_[406] 저널 Gene therapy: No improvement in outcomes with gene therapy for heart failure 2016-03
_[407] 저널 Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy 2013-08
_[408] 저널 Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome 2013-08
_[409] 뉴스 Gene therapy: 'Tame HIV' used to cure disease http://www.bbc.co.uk[...] BBC News, Health 2015-04-21
_[410] 저널 An emerging era of clinical benefit from gene therapy 2015-04
_[411] 뉴스 'Bubble kid' success puts gene therapy back on track https://www.newscien[...] The New Scientist 2013-10-30
_[412] 뉴스 Gene therapy cure for children with 'bubble baby' disease https://www.scienced[...] Science Daily 2014-11-18
_[413] 뉴스 Gene therapy provides safe, long-term relief for patients with severe hemophilia B https://www.scienced[...] Science Daily 2014-11-20
_[414] 저널 Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial 2014-03
_[415] 뉴스 Gene therapy restores sight in people with eye disease https://www.newscien[...] The New Scientist 2014-01-25
_[416] 저널 Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV https://archive.org/[...] 2014-03
_[417] 웹사이트 Scientists Create Genetically Modified Cells That Protect Against HIV http://io9.com/scien[...] 2014-03-06
_[418] ClinicalTrialsGov Stem Cell Gene Therapy for Sickle Cell Disease
_[419] ClinicalTrialsGov Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
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_[422] 뉴스 Protection Without a Vaccine https://www.nytimes.[...] 2015-03-09
_[423] 저널 AAV-expressed eCD4-Ig provides durable protection from multiple SHIV challenges 2015-03
_[424] 뉴스 Scientists Seek Ban on Method of Editing the Human Genome https://www.nytimes.[...] 2015-03-19
_[425] 뉴스 A Powerful New Way to Edit DNA https://www.nytimes.[...] 2015-03-03
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_[428] 뉴스 Baby girl is first in the world to be treated with 'designer immune cells' https://www.theguard[...] The Guardian 2015-11-05
_[429] 뉴스 Leukaemia cure hopes rise as girl is gene‑edited http://www.thetimes.[...] 2017-02-19
_[430] 뉴스 Scientists Place Moratorium on Edits to Human Genome That Could Be Inherited https://www.nytimes.[...] 2015-12-03
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_[432] 웹인용 Summary of opinion1 (initial authorisation) Strimvelis http://www.ema.europ[...] 2016-04-01
_[433] 뉴스 Europe gives green light to first gene therapy for children http://uk.reuters.co[...] 2016-04-01
_[434] 웹인용 Second gene therapy wins approval in Europe http://www.bionews.o[...] 2016-06-06
_[435] 뉴스 Gene Therapy Approved https://www.newscien[...] The New Scientist 2016-04-09
_[436] 저널 Chinese scientists to pioneer first human CRISPR trial http://www.nature.co[...] 2016-07
_[437] 뉴스 Chinese Scientists Become First to Use CRISPR Gene-Editing on Humans http://www.popularme[...] 2016-11-15
_[438] 저널 Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis-related lung disease 2016-06
_[439] 뉴스 New gene therapy ‘shrinks tumours like ice cubes’ http://www.thetimes.[...] 2017-03-01
_[440] 뉴스 Gene therapy ‘cures’ boy of blood disease that affects millions https://www.newscien[...] 2017-03
_[441] 웹인용 FDA approval brings first gene therapy to the United States https://www.fda.gov/[...] U.S. Food and Drug Administration 2017-08-30
_[442] 웹사이트 Approved Drugs - FDA approves axicabtagene ciloleucel for large B-cell lymphoma https://www.fda.gov/[...] 2018-01-05
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_[445] 저널 A Cure for Hemophilia within Reach http://www.nejm.org/[...] 2017-12
_[446] 웹인용 FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss https://www.fda.gov/[...] 2017-12-19
_[447] 뉴스 Spark Therapeutics Sets Price Of Blindness-Treating Gene Therapy At $850,000 https://www.forbes.c[...] 2018-01-03
_[448] 뉴스 The FDA approved a gene therapy to treat blindness in a groundbreaking moment for DNA-based medicine http://www.newsweek.[...] 2017-12-19
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_[458] 저널 US science advisers outline path to genetically modified babies http://www.nature.co[...] 2017-02-14
_[459] 저널 CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes 2015-05
_[460] 뉴스 Chinese Scientists Edit Genes of Human Embryos, Raising Concerns https://www.nytimes.[...] 2015-04-24
_[461] 웹인용 Scientists OK genetically engineering babies https://nypost.com/2[...] Reuters 2017-02-17
_[462] 뉴스 Human Gene Editing Receives Science Panel’s Support https://www.nytimes.[...] 2017-02-17
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